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2 / April 2020 © Informa UK Ltd 2020 (Unauthorized photocopying prohibited.)

Executive SummaryThis is an update of recommendations from the European Medicines Agency’s Committee for Medicinal Products for Human Use on the authorization of new medicines in the European Union, and updates on EU marketing authorization changes recommended by the CHMP.

The European Medicines Agency’s key scientific panel, the Committee for Medicinal Products for Human Use (CHMP), meets once a month and among other things adopts positive or negative opinions on marketing authorization applications (MAAs) filed through the centralized procedure for new molecular entities (NMEs), biosimilars and generic medicines. CHMP opinions are sent to the European Commission, which makes a final,

legally binding decision, usually within 67 days.The CHMP at its monthly plenary meetings also assesses requests for modifications or extensions to existing marketing authorizations.

The table below provides an overview of decisions on new marketing applications, applications to extend the indication of existing marketing authorizations and requests for the re-examination of negative opinions the CHMP has adopted. It also covers the withdrawal of MAAs by companies.

The table has now been updated with products that the CHMP discussed at its meeting in February 2020.

Please note: Data in the following table is a sample only.

European CHMP Opinions and MAA Updates

Company Brand and Generic Name Indication Announce-

ment Date Recommendation/ Update

Bristol-Myers Squibb

Opdivo/Yervoy (nivolumab/ipilimumab)

Extension of indication to include the use of Opdivo and Yervoy in the treatment of metastatic non-small cell lung cancer that has not been treated previously

Feb-20

Application withdrawn by company. BMS said it withdrew its application based on the fact that the EMA could not conclude that there was a positive benefit-risk balance for the medicines in the treatment of non-small cell lung cancer that has not been treated previously

Blue Earth Diagnostics

Axumin (fluciclovine (18F))

Extension of indication to include use in the diagnosis of glioma and the continuing assessment of the disease

Feb-20

Application withdrawn by company. Blue Earth said it withdrew its application because the EMA considered that the data provided did not allow it to determine that the medicine’s benefit outweighs its risk when used for detecting glioma


Eli Lilly Emgality (galcanezumab)

Extension of indication to include prevention of attacks in adults who suffer from episodic cluster headache. For this use, Emgality was to be available as a pre-filled syringe containing 100mg of the medicine.

Feb-20 Negative opinion, extension

Takeda Entyvio (vedolizumab)

Addition of a new pharmaceutical form: a 108mg solution for injection to be given subcutaneously. The new formulation is to be used as maintenance treatment for all the authorized indications of Entyvio - ie Crohn’s disease and ulcerative colitis

Feb-20 Positive opinion, extension

Amgen Otezla (apremilast)

Extension of indication to include use as a treatment of adult patients with oral ulcers associated with Behçet’s disease who are candidates for systemic therapy


Boehringer Ingelheim Ofev (nintedanib)

Extension of indication to include use as a treatment of systemic sclerosis associated interstitial lung disease in adults


Takeda Alunbrig (brigatinib)

Extension of indication to include use as monotherapy for the treatment of adult patients with anaplastic lymphoma kinase (ALK)-positive advanced non small cell lung cancer that has not previously been treated with an ALK inhibitor.


Accord Healthcare Tigecycline Accord (tigecycline)

Treatment of complicated skin and soft tissue infections and complicated intra-abdominal infections

Feb-20 Positive opinion

Shionogi Fetcroja (cefiderocol)

Treatment of infections due to aerobic Gram-negative organisms in adults with limited treatment options

Feb-20 Positive opinion

Merck Sharp & Dohme

Keytruda (pembrolizumab)

Treatment of cancer of the oesophagus Jan-20

Application withdrawn by the company. Merck said it decided to withdraw the application because the results of the study were not considered sufficient to support an extension of indication at this time

Celgene Idhifa (enasidenib) Treatment of adults with acute myeloid leukemia Jan-20

Application withdrawn by company. Celgene said it decided to withdraw the application because it could not fully address the major objections raised by the EMA


AbbVie Venclyxto (venetoclax)

Extension of indication for use in combination with obinutuzumab for the treatment of adult patients with previously untreated chronic lymphocytic leukemia

Jan-20 Positive opinion, extension

Gilead Sciences Tybost (cobicistat)

Extension of indication to expand use of Tybost from use in adults with HIV to use in adolescents aged 12 years and older – weighing at least 35kg co administered with atazanavir, or weighing at least 40kg co administered with darunavir


SanofiSuliqua (insulin glargine/lixisenatide)

The indication for Suliqua has been changed. It now reads that the product is indicated for the treatment of adults with insufficiently controlled type 2 diabetes mellitus to improve glycemic control as an adjunct to diet and exercise in addition to metformin with or without sodium-glucose co-transporter-2 (SGLT-2) inhibitors


Janssen-Cilag Rezolsta (darunavir/cobicistat)

Extension of indication to expand use of Rezolsta, in combination with other antiretroviral products, for use in the treatment of HIV in adults to use in adolescents (aged 12 years and older, weighing at least 40kg)


Roche MabThera (rituximab)

Two new extensions of indication: MabThera, in combination with glucocorticoids, is indicated for the induction of remission in pediatric patients (aged ≥2 to <18 years old) with severe, active granulomatosis with polyangiitis (Wegener’s) and microscopic polyangiitis; and MabThera, in combination in combination with chemotherapy is indicated for the treatment of pediatric patients (aged ≥6 months to <18 years old) with previously untreated advanced stage CD20 positive diffuse large B-cell lymphoma, Burkitt lymphoma/Burkitt leukemia (mature B-cell acute leukemia) or Burkitt-like lymphoma


Biofrontera Bioscience

Ameluz (5-aminolevulinic acid)

Indication when used to treat mild to moderate actinic keratoses extended to use on the extremities and trunk/neck



SciPharmTrepulmix (treprostinil sodium)

Treatment of chronic thromboembolic pulmonary hypertension

Jan-20 Positive opinion

Teva Pharmaceutical

Budesonide/Formoterol Teva Pharma (budesonide/formoterol fumarate dihydrate)

Treatment of asthma and chronic obstructive pulmonary disease Jan-20 Positive opinion

Accord Healthcare Cinacalcet Accordpharma (cinacalcet)

Treatment of secondary hyperparathyroidism, parathyroid carcinoma and primary hyperparathyroidism


Mylan Azacitidine Mylan (azacitidine)

Treatment of myelodysplastic syndromes, chronic myelomonocytic leukemia and acute myeloid leukemia


betapharm Arzneimittel

Azacitidine betapharm (azacitidine)

Treatment of myelodysplastic syndromes, chronic myelomonocytic leukemia and acute myeloid leukemia


MylanArsenic trioxide Mylan (arsenic trioxide)

Treatment of acute promyelocytic leukemia Jan-20 Positive opinion

Pfizer Ruxience (rituximab)

Treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and pemphigus vulgaris


Pfizer Staquis (crisaborole) Treatment of atopic dermatitis Jan-20 Positive opinion

Bayer Nubeqa (darolutamide)

Treatment of men with non-metastatic castration-resistant prostate cancer (nmCRPC) who are at high risk of developing metastatic disease


Esperion Therapeutics

Nustendi (bempedoic acid/ezetimibe)

Treatment of primary hypercholesterolemia and mixed dyslipidemia


Esperion Therapeutics

Nilemdo (bempedoic acid)

Treatment of adults with primary hypercholesterolemia (heterozygous familial and non-familial) or mixed dyslipidemia


Eli Lilly Liumjev (insulin lispro)

Treatment of diabetes mellitus in adults Jan-20 Positive opinion

Emergent BioSolutions

Vaxchora (Cholera vaccine (recombinant, live, oral))

For prophylaxis against cholera Jan-20 Positive opinion


Novo Nordisk Rybelsus (semaglutide)

Treatment of adults with insufficiently controlled type 2 diabetes to improve glycemic control as an adjunct to diet and exercise


Alnylam Pharmaceuticals Givlaari (givosiran)

Treatment for acute hepatic porphyria in adults and adolescents aged 12 years and older


Pfizer Vyndaqel (tafamidis)

Extension of indication to include a new presentation of Vyndaqel 61mg soft capsules for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)

Dec-19 Positive opinion, extension

Janssen-Cilag Stelara (ustekinumab)

Extension of indication for use in younger patients when used to treat pediatric plaque psoriasis. The new wording now says Stelera is indicated for the treatment of moderate to severe plaque psoriasis in children and adolescent patients from the age of 6 years and older, who are inadequately controlled by, or are intolerant to, other systemic therapies or phototherapies


Janssen-Cilag Sirturo (bedaquiline)

Extension of indication to use in adolescents. The new wording now says Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug-resistant tuberculosis in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability


Janssen-Cilag Erleada (apalutamide)

Extension of indication to use in adult men for the treatment of metastatic hormone-sensitive prostate cancer in combination with androgen deprivation therapy



Astellas Pharma Dificlir (fidaxomicin)

Extension of indication for the film-coated tablets. Dificlir film-coated tablets are still indicated in adults but the wording now says they are indicated for the treatment of Clostridioides difficile infections (CDI) also known as C. difficile-associated diarrhea (CDAD) and that they can also be used to treat pediatric patients with a body weight of at least 12.5kg


Janssen-Cilag Darzalex (daratumumab)

Extension of indication for use of Darzalex in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant


Eli Lilly Cyramza (ramucirumab)

Extension of indication for the use of Cyramza in combination with erlotinib for the first-line treatment of adult patients with metastatic non-small cell lung cancer with activating epidermal growth factor receptor (EGFR) mutations


Helsinn Birex Pharmaceuticals

Akynzeo (fosnetupitant/palonosetron)

A new pharmaceutical form associated with a new strength and a new route of administration as follows: Akynzeo 235mg/0.25mg powder for concentrate for solution for infusion for intravenous use


Accord HealthcareDexmedetomidine Accord (dexmedetomidine)

For the induction of light to moderate sedation of adults in an intensive care unit

Dec-19 Positive opinion

Accord Healthcare Azacitidine Accord (azacitidine)

Treatment of myelodysplastic syndromes, chronic myelomonocytic leukemia and acute myeloid leukemia.


Pfizer Amsparity (adalimumab)

Treatment of certain inflammatory and autoimmune disorders Dec-19 Positive opinion

Merck Sharp & Dohme

Recarbrio (imipenem/cilastatin/relebactam)

Treatment of infections due to aerobic Gram-negative organisms in adults with limited treatment options


Novartis Beovu (brolucizumab)

Treatment of neovascular (wet) age-related macular degeneration Dec-19 Positive opinion

D&A Pharma Hopveus (sodium oxybate) Treatment of alcohol dependence Nov-19

Re-examination of October 2019 negative opinion requested by the company


Kiadis Pharma Luxceptar (viable T-cells)

Treatment of patients with blood cancers who are receiving a type of blood stem cell transplant

Nov-19

Withdrawn by the company. Kiadis said it decided to withdraw its application because of the Agency’s view that the current evidence was not sufficient to conclude on the effectiveness of treatment

Aradigm Pharmaceuticals

Linhaliq (ciprofloxacin)

For treating and preventing flare-ups of bronchiectasis in patients with long-term lung infection caused by Pseudomonas aeruginosa bacteria

Nov-19

Withdrawn by company. Aradigm said it decided to withdraw its application because the data provided did not allow the CHMP to conclude on a positive benefit-risk balance for the medicine

Celgene Revlimid (lenalidomide)

Extension of indication to include use in combination with rituximab (anti-CD20 antibody) for the treatment of adult patients with previously treated follicular lymphoma (Grade 1 – 3a)

Nov-19 Positive opinion, extension

RocheKadcyla (trastuzumab emtansine)

Extension of indication to include use as a single agent for the adjuvant treatment of adult patients with HER2-positive early breast cancer who have residual invasive disease, in the breast and/or lymph nodes, after neoadjuvant taxane-based and HER2-targeted therapy

Nov-19 Positive opinion, extension

Accord Healthcare Deferasirox Accord (deferasirox)

Treatment of chronic iron overload due to blood transfusions in patients with beta thalassemia and other anemias

Nov-19 Positive opinion

Mylan

Clopidogrel/Acetylsalicylic acid Mylan (clopidogrel / acetylsalicylic acid)

Secondary prevention of atherothrombotic events Nov-19 Positive opinion

Rigel Pharmaceuticals

Tavlesse (fostamatinib)

Treatment of primary immune thrombocytopenia Nov-19 Positive opinion

Jazz Pharmaceuticals

Sunosi (solriamfetol)

Treatment of excessive daytime sleepiness in patients with narcolepsy and obstructive sleep apnea


Roche Polivy (polatuzumab vedotin)

Treatment of relapsed/refractory diffuse large B-cell lymphoma Nov-19 Positive opinion, conditional,

further data will be required

Novartis Mayzent (siponimod)

Treatment of adult patients with secondary progressive multiple sclerosis with active disease


Novartis Isturisa (osilodrostat) Treatment of Cushing’s syndrome Nov-19 Positive opinion

Emmaus Life Sciences Xyndari (glutamine) Treatment of sickle cell disease Oct-19

Withdrawn by company. Emmaus said the withdrawal is based on a change in the company’s marketing strategy


Paratek Pharmaceuticals

Nuzyra ( omadacycline)

Treatment of community-acquired bacterial pneumonia and bacterial infections of the skin and skin structures

Oct-19

Withdrawn by company. Paratek said it decided to withdraw its application because it would not be commercially feasible to market Nuzyra just for the treatment of skin and skin structure infections

Sun Pharmaceutical Industries

Ekesivy (diclofenamide)

Treatment of muscle disorders called periodic paralysis Oct-19

Withdrawn by company. Sun said it decided to withdraw its application because it could not address the agency’s concerns within the available time frame

PTC Therapeutics Translarna (ataluren)

Extension of indication to add the treatment of patients with Duchenne muscular dystrophy who are no longer able to walk

Oct-19Negative opinion, on re-examination by CHMP, indication not extended

Novartis Revolade (eltrombopag)

Extension of indication to add treatment of previously untreated patients with severe aplastic anemia from 12 years of age who cannot have stem cell transplantation

Oct-19Negative opinion, on re-examination by CHMP, indication not extended

Sanofi Toujeo (insulin glargine)

Indication extended to include treatment of diabetes mellitus in adolescents and children from the age of six years

Oct-19 Positive opinion, extension

Merck Sharp & Dohme


Extension of indication for use as monotherapy or in combination with platinum and 5-fluorouracil (5-FU) chemotherapy for the first-line treatment of metastatic or unresectable recurrent head and neck squamous cell carcinoma in adults whose tumors express PD-L1 with a CPS ≥ 1


Vertex Pharmaceuticals Kalydeco (ivacaftor)

Extension of indication to include use in infants aged at least six months, toddlers and children weighing 5kg to less than 25kg with cystic fibrosis who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R


Janssen-Cilag Darzalex (daratumumab)

Extension of indication to include use in combination with lenalidomide and dexamethasone or with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant



Daiichi Sankyo Vanflyta (quizartinib)

Treatment of adults with acute myeloid leukemia Oct-19 Negative opinion

D&A Pharma Hopveus (sodium oxybate)

Treatment of alcohol dependence in patients in whom other treatments are not effective or cannot be used

Oct-19 Negative opinion

UCB Pharma Evenity (romosozumab)

Treatment of postmenopausal women with severe osteoporosis at high risk of fracture

Oct-19 Positive opinion, confirmed on re-examination by CHMP

Mundipharma Biologics

Pegfilgrastim Mundipharma (pegfilgrastim)

For reducing the duration of neutropenia and the incidence of febrile neutropenia due to chemotherapy

Oct-19 Positive opinion

Janssen-Cilag Spravato (esketamine)

For combination treatment in adults with treatment-resistant major depressive disorder


AbbVie Rinvoq (upadacitinib) Treatment of rheumatoid arthritis Oct-19 Positive opinion

Menarini Group Quofenix (delafloxacin)

Treatment of acute bacterial skin and skin structure infections in adults when it is considered inappropriate to use other antibacterial agents


Eli Lilly Baqsimi (glucagon)Treatment of severe hypoglycemia in patients with diabetes aged four years and older


Merck Sharp & Dohme

Ervebo (Ebola Zaire Vaccine (rVSVΔG-ZEBOV-GP, live)

For active immunization of individuals aged 18 years and older at risk of infection with the Ebola virus

Oct-19 Positive opinion, conditional, further data will be required

Emmaus Life Sciences Xyndari (glutamine) Treatment of sickle cell disease Sep-19 Withdrawn by the company

Lilly Trulicity (dulaglutide)

The indication for Trulicity has been changed - the wording now says that Trulicity is indicated for the treatment of adults with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise: (i) as monotherapy when metformin is considered inappropriate due to intolerance or contraindications; (ii) in addition to other medicinal products for the treatment of diabetes

Sep-19 Positive opinion, extension

Aventis Pharma Taxotere (docetaxel)

Extension of indication to include use in combination with androgen-deprivation therapy, with or without prednisone or prednisolone, for the treatment of patients with metastatic hormone-sensitive prostate cancer



Celltrion Healthcare

Remsima (infliximab)

Inclusion of a new route of administration, strength and pharmaceutical form: Remsima 120mg solution for injection for subcutaneous use in pre-filled pens or syringes (Remsima for subcutaneous use is authorized only in the rheumatoid arthritis indication)


Novartis Lucentis (ranibizumab)

Extension of indication to include use as a treatment for proliferative diabetic retinopathy


Sanofi Dupixent (dupilumab)

Extension of indication to include use as an add-on therapy with intranasal corticosteroids for the treatment of adults with severe chronic rhinosinusitis with nasal polyposis for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control


Zentiva Docetaxel Zentiva (docetaxel)

Extension of indication to include use in combination with prednisone or prednisolone for the treatment of patients with hormone refractory metastatic castration-resistant prostate cancer


GlaxoSmithKline Benlysta (belimumab)

The indication for Benlysta has been changed - it is still indicated for use in adults but now the wording says it can be used as add-on therapy in patients aged 5 years and older with active, autoantibody-positive systemic lupus erythematosus with a high degree of disease activity (eg, positive anti dsDNA and low complement) despite standard therapy


Merck Bavencio (avelumab)

Extension of indication to use in combination with axitinib for the first-line treatment of adult patients with advanced renal cell carcinoma


Orphelia Pharma Ivozall (clofarabine) Treatment of acute lymphoblastic leukemia in pediatric patients Sep-19 Positive opinion

Fresenius KabiBortezomib Fresenius Kabi (bortezomib)

Treatment of multiple myeloma and mantle cell lymphoma Sep-19 Positive opinion

Accord HealthcareArsenic trioxide Accord (arsenic trioxide)

Treatment of acute promyelocytic leukemia Sep-19 Positive opinion

Plethora Pharma Solutions

Senstend (lidocaine/prilocaine)

Treatment of premature ejaculation in adult men Sep-19 Positive opinion, informed

consent


Aerie Pharmaceuticals

Rhokiinsa (netarsudil)

Treatment of patients with glaucoma or ocular hypertension Sep-19 Positive opinion

AstraZeneca

Qtrilmet (metformin hydrochloride/saxagliptin/dapagliflozin)

Treatment of type 2 diabetes mellitus Sep-19 Positive opinion

Astellas Pharma Xospata (gilteritinib)

Treatment of adult patients who have relapsed or refractory acute myeloid leukemia with a FLT3 mutation

Sep-19 Positive opinion

PTC Therapeutics Translarna (ataluren)

Extension of indication to add the treatment of patients with Duchenne muscular dystrophy who can no longer walk

Jul-19Re-examination of June 2019 negative opinion requested by the company

Novartis Revolade (eltrombopag)

Extension of indication to include treatment of previously untreated patients with severe aplastic anemia from 2 years of age

Jul-19Re-examination of June 2019 negative opinion requested by the company

UCB Pharma Evenity (romosozumab) Treatment of osteoporosis Jul-19

Re-examination of June 2019 negative opinion requested by the company

Merck Sharp & Dohme

Zerbaxa (ceftolozane/tazobactam)

Extension of indication to include treatment of hospital-acquired pneumonia, including ventilator-associated pneumonia

Jul-19 Positive opinion, extension

Roche Tecentriq (atezolizumab)

Extension of indication to include use in combination with nab paclitaxel and carboplatin for the first-line treatment of adult patients with metastatic non-squamous non-small cell lung cancer who do not have EGFR mutant or ALK positive NSCLC. Also, extension of indication to include use in combination with carboplatin and etoposide for the first-line treatment of adult patients with extensive-stage small cell lung cancer.


Janssen-Cilag Stelara (ustekinumab)

Extension of indication to include the treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic or have medical contraindications to such therapies


Alexion Pharmaceuticals Soliris (eculizumab)

Extension of indication to include treatment of neuromyelitis optica spectrum disorder in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease



Novartis Lucentis (ranibizumab)

Extension of indication to use in preterm infants for the treatment of retinopathy of prematurity (ROP) with zone I (stage 1+, 2+, 3 or 3+), zone II (stage 3+) or AP-ROP (aggressive posterior ROP) disease


Les Laboratoires Servier

Lonsurf (trifluridine/tipiracil)

Extension of indication to use as monotherapy for the treatment of adult patients with metastatic gastric cancer including adenocarcinoma of the gastroesophageal junction, who have been previously treated with at least two prior systemic treatment regimens for advanced disease. The existing indication for colorectal cancer has also been changed to use as monotherapy for the treatment of adult patients with metastatic colorectal cancer who have been previously treated with, or are not considered candidates for, available therapies including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-VEGF agents, and anti EGFR agents


Merck Sharp & Dohme


Extension of indication to include use in combination with axitinib for the first-line treatment of advanced renal cell carcinoma in adults


Bristol-Myers Squibb

Empliciti (elotuzumab)

Extension of indication to include use in combination with pomalidomide and dexamethasone for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on the last therapy


Mylan Deferasirox Mylan (deferasirox)

Treatment of chronic iron overload due to blood transfusions in patients with beta thalassemia major, non-transfusion-dependent thalassemia syndromes and other anemias

Jul-19 Positive opinion

Theratechnologies Trogarzo (ibalizumab) Treatment of HIV infection Jul-19 Positive opinion

Acorda Therapeutics Inbrija (levodopa) Treatment of symptoms of off

periods in Parkinson’s disease Jul-19 Positive opinion


GW Pharma Epidyolex (cannabidiol)

Treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome

Jul-19 Positive opinion

Bayer Vitrakvi (larotrectinib)

Treatment of patients with solid tumors that display a neurotrophic tyrosine receptor kinase (NTRK) gene fusion

Jul-19 Positive opinion, conditional, further data will be required


New EU Approvals

Executive SummaryThe Pink Sheet’s list of EU centralized approvals of new active substances has been updated with three new products including Novartis’s Beovu for treating neovascular (wet) age related macular degeneration. The list, which contains information dating back to January 2018, includes brand name, generic name, company, therapeutic indication, date of marketing authorization and product type (eg, medicine, vaccine, biologic).

The Pink Sheet’s regularly updated list of products containing new active substances that have been approved for marketing in the EU under the centralized authorization procedure now includes three new products.

The great majority of new, innovative medicines and vaccines that are marketed in the EU go

through the centralized procedure. The European Medicines Agency’s Committee for Medicinal Products for Human Use (the CHMP) carries out a scientific assessment of the initial marketing authorization application (MAA) and gives a recommendation on whether the medicine should be marketed or not. CHMP recommendations are forwarded to the European Commission for a legally binding decision.

Once granted, centralized marketing authorizations are valid in all EU member states as well as in the European Economic Area countries of Iceland, Liechtenstein and Norway.

The list is updated regularly with new approvals and is current as of 20 February 2020. It includes information dating back to January 2018.


Company Brand/Generic Name IndicationDate of Marketing Authorization Announcement

Product Type/Status

AstraZeneca Fasenra (benralizumab)

Respiratory - maintenance treatment for severe eosinophilic asthma

8-Jan-18 Biologic

Roche Ocrevus (ocrelizumab) CNS - multiple sclerosis 8-Jan-18 Biologic

Shire Pharmaceuticals Adynovi (rurioctocog alfa pegol) Blood disorders - hemophilia A 8-Jan-18 Biologic

Merck Sharp & Dohme Prevymis (letermovir)

Infectious diseases - prophylaxis of cytomegalovirus reactivation and disease in adult CMV-seropositive recipients of allogeneic hematopoietic stem cell transplant

8-Jan-18 Medicine, orphan

Novo Nordisk Ozempic (semaglutide) Metabolic - adults with insufficiently controlled type 2 diabetes 8-Feb-18 Medicine

Roche/Chugai Hemlibra (emicizumab) Blood disorders - hemophilia A 18-Feb-18 Biologic

Kyowa Kirin Crysvita (burosumab) Metabolic - X-linked hyperphosphatemia 19-Feb-18

Biologic, orphan, conditional approval


Merck Sharp & Dohme Steglatro (ertugliflozin)* Metabolic - type 2 diabetes 21-Mar-18 Medicine

GlaxoSmithKline Biologicals

Shingrix (recombinant varicella zoster vaccine)

Infectious diseases - prevention of herpes zoster/post-herpetic neuralgia in adults 50 years and over

21-Mar-18 Vaccine

AstraZeneca Lokelma (sodium zirconium cyclosilicate)

Metabolic - oral potassium removing agent for hyperkalemia 22-Mar-18 Medicine

Merck Sharp & Dohme Steglujan (ertugliflozin/sitagliptin) Metabolic - type 2 diabetes 23-Mar-18 Medicne

Tigenix/Takeda Alofisel (darvadstrocel)

Immunology/Gastrointestinal - an allogeneic stem cell therapy for complex perianal fistulas in Crohn’s disease

23-Mar-18

Advanced therapy medicinal product, orphan

Merck Sharp & Dohme

Segluromet (ertugliflozin/metformin hydrochloride)

Metabolic - type 2 diabetes 23-Mar-18 Medicine

Chiesi Farmaceutici Lamzede (velmanase alfa)

Genetic disorders - alpha-mannidosis 23-Mar-18

Biologic, orphan, exceptional circumstances approval

BioCryst UK Alpivab (peramivir)Infectious diseases - uncomplicated influenza in adults and children from two years of age

13-Apr-18 Medicine

Pfizer Mylotarg (gemtuzumab ozogamicin)

Cancer - acute myeloid leukemia 19-Apr-18 Biologic, orphan

Clovis Oncology Rubraca (rucaparib) Cancer - ovarian neoplasms 24-May-18 Medicine, orphan

Gilead Sciences

Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) - bictegravir is the new active substance

Infectious diseases - HIV infection in adults without present or past evidence of viral resistance to the integrase inhibitor class, emtricitabine or tenofovir

21-Jun-18 Medicine

Akcea Therapeutics (Ionis Pharmaceuticals) Tegsedi (inotersen)

Neurologic - for treatment of stage 1 or stage 2 polyneuropathy in patients with hereditary transthyretin amyloidosis (hATTR)

6-Jul-18

Medicine, orphan, accelerated assessment

Otsuka Rxulti (brexpiprazole) Neurologic - schizophrenia in adults 26-Jul-18 Medicine

Novartis Aimovig (erenumab)Analgesics - for prophylaxis of migraine in adults who have at least 4 migraine days per month

26-Jul-18 Biologic

Aegerion Pharmaceuticals Myalepta (metreleptin)

Alimentary tract and metabolism - an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients

30-Jul-18 Biologic, orphan


Novartis Kymriah (tisagenlecleucel)

Cancer - pediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukemia that is refractory, in relapse post transplant or in second or later relapse. Adult patients with relapsed or refractory diffuse large B cell lymphoma after two or more lines of systemic therapy

23-Aug-18

Advanced therapy medicinal product, orphan, accelerated assessment

Kite Pharma Yescarta (axicabtagene ciloleucel)

Cancer - adults with relapsed or refractory diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy

23-Aug-18

Advanced therapy medicinal product, orphan

Ultragenyx Mepsevii (vestronidase alfa)

Metabolism - non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome)

23-Aug-18

Biologic, orphan, exceptional circumstances approval

Alnylam Onpattro (patisiran)

Neurologic - hereditary transthyretin-mediated (hATTR) amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy

27-Aug-18

Medicine, orphan, accelerated assessment

Puma Biotechnology Nerlynx (neratinib)

Cancer - extended adjuvant treatment of adult patients with early stage hormone receptor positive HER2-overexpressed/amplified breast cancer and who are less than one year from the completion of prior adjuvant trastuzumab based therapy

31-Aug-18 Medicine

Ablynx/Sanofi Cablivi (caplacizumab)

Antithrombotic - adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression

31-Aug-18 Biologic, orphan

Almirall Ilumetri (tildrakizumab)

Immunology, Dermalology - adults with moderate to severe plaque psoriasis who are candidates for systemic therapy

17-Sep-18 Biological

Pierre Fabre Médicament Mektovi (binimetinib)

Cancer - for use in combination with encorafenib for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation

20-Sep-18 Medicine

Pierre Fabre Médicament Braftovi (encorafenib)

Cancer - for use in combination with binimetinib for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation

20-Sep-18 Medicine

Tetraphase Pharmaceuticals Xerava (eravacycline) Infectious disease - complicated

intra-abdominal infections in adults 20-Sep-18 Medicine


AstraZeneca Imfinzi (durvalumab)

Cancer - for use as monotherapy for the treatment of locally advanced, unresectable non-small cell lung cancer in adults whose tumors express PD-L1 on ≥ 1% of tumor cells and whose disease has not progressed following platinum based chemoradiation therapy

21-Sep-18 Biologic

Eli Lilly Verzenios (abemaciclib)

Cancer - treatment of women with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative locally advanced or metastatic breast cancer in combination with an aromatase inhibitor or fulvestrant as initial endocrine-based therapy, or in women who have received prior endocrine therapy

27-Sep-18 Medicine

Vertex Pharmaceuticals

Symkevi (tezacaftor/ivacaftor)

Genetic disorders - Indicated for use in a combination regimen with ivacaftor 150mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A�G, S945L, S977F, R1070W, D1152H, 2789+5G�A, 3272 26A�G, and 3849+10kbC�T

31-Oct-18 Medicine, orphan

Rempex Pharmaceuticals

Vabomere (meropenem/vaborbactam)

Infectious diseases -treatment of the following infections in adults: complicated urinary tract infection (cUTI), including pyelonephritis; complicated intra-abdominal infection (cIAI); hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP). Treatment of patients with bacteremia that occurs in association with, or is suspected to be associated with, any of the infections listed above. Also for the treatment of infections due to aerobic Gram-negative organisms in adults with limited treatment options

20-Nov-18 Medicine

Shire Takhzyro (lanadelumab)

Genetic disorders - for routine prevention of recurrent attacks of hereditary angioedema in patients aged 12 years and older

22-Nov-18

Biologic, orphan, accelerated assessment


Spark Therapeutics Luxturna (voretigene neparvovec)

Ophthalmic - treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells

22-Nov-18

Advanced therapy medicinal product, orphan drug

Takeda Alunbrig (brigatinib)

Cancer - for use as a monotherapy for the treatment of adult patients with anaplastic lymphoma kinase-positive advanced non-small cell lung cancer previously treated with crizotinib

22-Nov-18 Medicine

Kyowa Kirin Poteligeo (mogamulizumab)

Infectious diseases - treatment of adult patients with mycosis fungoides or Sézary syndrome who have received at least one prior systemic therapy

22-Nov-18 Biologic, orphan

Bayer Jivi (damoctocog alfa pegol)

Blood disorders - treatment and prophylaxis of bleeding in previously treated patients ≥ 12 years of age with hemophilia A (congenital factor VIII deficiency)

22-Nov-18 Biologic, orphan drug

Merck Sharp & DohmeDelstrigo (doravirine/lamivudine/ tenofovir disoproxil)

Infectious diseases - treatment of adults infected with HIV 1 without past or present evidence of resistance to the NNRTI class, lamivudine, or tenofovir

22-Nov-18 Medicine

Merck Sharp & Dohme Pifeltro (doravirine)

Infectious diseases - for use in combination with other antiretroviral medicinal products, for the treatment of adults infected with HIV 1 without past or present evidence of resistance to the NNRTI class

22-Nov-18 Medicine

SanofiDengvaxia (dengue tetravalent vaccine (live, attenuated))

Indicated for the prevention of dengue disease caused by dengue virus serotypes 1, 2, 3 and 4 in individuals 9 to 45 years of age with prior dengue virus infection and living in endemic areas

12-Dec-18 Vaccine


New Filings At The EMA

Executive SummaryNew medicines under evaluation at the European Medicines Agency.

This is a regularly updated list of products that have been filed with the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) under the EU’s centralized authorization procedure.

The information is based on the EMA’s monthly updated list of medicines under review at the agency, CHMP monthly meeting agendas, company press releases, and original research by the Pink Sheet. The latest list from the EMA was compiled on 7 February. (Also see “Potential Cardiovascular Blockbuster Inclisiran Among New Filings At EMA” - Pink Sheet, 14 Feb, 2020.)

The EMA does not identify in its own list the sponsors of marketing authorization applications that are under review. It gives only the international non-proprietary name (INN)

and therapeutic area. For generic and biosimilar medicines, it provides the INN (active moiety only, with no information on salt, ester or derivative) and therapeutic area. The CHMP monthly meeting agendas sometimes identify the name of sponsors of orphan drugs only.

The EMA only lists information on medicines whose applications have been validated at the time the agency’s list in question or CHMP agenda is compiled.

Information on CHMP opinions and on EU approvals can be found elsewhere in the EU Performance Tracker section of the Pink Sheet website.

Information on the different types of applications or approval pathways involved – for example, accelerated assessment, informed consent application, conditional approval – is provided in footnotes below the table.


Company INN Therapeutic area or indication

Date of EMA list compilation, CHMP agenda, or company press release

Product Type/Status

not known risperidone psycholeptic February 7 2020 generic

not known lenalidomide immunosuppressant February 7 2020 generic

not known doxorubicin antineoplastic medicine February 7 2020 generic

not known bevacizumab antineoplastic medicine February 7 2020 biosimilar

Celgenefedratinib (US brand name Inrebic)

treatment of myelofibrosis February 7 2020 medicine, orphan

GlaxoSmithKline belantamab mafodotin

treatment of relapsed or refractory multiple myeloma

February 7 2020medicine, orphan, accelerated assessment




Product Type/Status

Kite

KTE-X19 (autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured)

treatment of relapsed or refractory mantle cell lymphoma

February 7 2020ATMP, orphan, accelerated assessment

Seattle Genetics tucatinib

treatment of patients with locally advanced or metastatic HER2-positive breast cancer

February 7 2020 medicine

Lilly selpercatinib

treatment of RET-mutant medullary thyroid cancer, advanced RET fusion-positive non-small cell lung cancer and RET fusion-positive thyroid cancer


Roche

Perjeta (pertuzumab)/Herceptin (trastuzumab)

treatment of patients with HER2-positive breast cancer


Novartis ofatumumab treatment of relapsing forms of multiple sclerosis February 7 2020 medicine

Novartis inclisiran lipid modifying medicine February 7 2020 medicine

PTC TherapeuticsPTC-AADC (eladocagene exuparvovec)

treatment of aromatic L-amino acid decarboxylase (AADC) deficiency

January 13 2020 medicine, orphan

ViiV Healthcare fostemsavir

for use in combination with other antiretrovirals for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive antiviral regimen due to resistance, intolerance or safety considerations

January 10 2020medicine, accelerated assessment

not known glucagon pancreatic hormones January 6 2020 generic

not known azathioprine immunosuppressant January 6 2020 generic

Paion remimazolam for procedural sedation January 6 2020 medicine




Product Type/Status

Kyowa Kirin istradefylline (KW-6002)

adjunctive treatment to levodopa-based regimens in adult patients with Parkinson’s disease experiencing “off” time

January 6 2020 medicine

Verastem duvelisib (Copiktra)

treatment of patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma and relapsed or refractory follicular lymphoma


Aerie Pharmaceuticals

netarsudil and latanoprost (Roclanda)

for reducing elevated intraocular pressure in patients with glaucoma

January 6 2020 medicine

Incyte pemigatinib

treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that is relapsed or refractory after at least one line of systemic therapy


Innate Pharmamoxetumomab pasudotox (Lumoxiti)

treatment of adults with relapsed or refractory hairy cell leukemia who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog


BioMarin Pharmaceutical

valoctocogene roxaparvovec

treatment of adults with severe hemophilia A January 6 2020

medicine, accelerated assessment, orphan

Advicenne Pharma

potassium citrate/potassium hydrogen carbonate

treatment of distal renal tubular acidosis (dRTA) in patients aged six months and older

December 9 2019 medicine, orphan

Novartis

QVM149 (indacaterol, glycopyrronium and mometasone)

asthma December 2 2019 medicine

Amarinicosapent ethyl (Vascepa - US name)

cardiovascular risk reduction December 2 2019 medicine

Roche baloxavir marboxil (Xofluza - US name) treatment of flu December 2 2019 medicine




Product Type/Status

Orchard Therapeutics

OTL-200 (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase a gene)

gene therapy for metachromatic leukodystrophy

December 2 2019ATMP, orphan, accelerated assessment

Janssen Ad26.ZEBOV Ebola Zaire vaccine November 7 2019vaccine, accelerated assessment

Janssen MVA-BN-Filo Ebola Zaire vaccine November 7 2019vaccine, accelerated assessment

not known

fluticasone (propionate)/salmeterol (xinafoate)

treatment of asthma November 4 2019 medicine

Sanofiinfluenza quadrivalent vaccine (rDNA)

influenza vaccine November 4 2019 vaccine

Sanofi

Meningococcal group A, C, W135 and Y conjugate vaccine

immunization against Neisseria meningitidis serogroups A, C, W-135 and Y

November 4 2019 vaccine

not known caffeine citrate treatment of primary apnoea November 4 2019 generic

Biocon/Mylan insulin aspart treatment of diabetes mellitus November 4 2019 biosimilar

not known sunitinib

treatment of gastrointestinal stromal tumor and metastatic renal cell carcinoma and pancreatic neuroendocrine tumors

November 4 2019 generic

AstraZeneca acalabrutinib

treatment of adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma

November 4 2019 medicine, orphan

MYR Pharmaceuticals

bulevirtide (acetate)

treatment of chronic hepatitis delta virus (HDV) infection in adult patients with compensated liver disease

November 4 2019medicine, orphan, accelerated assessment




Product Type/Status

Vertex Pharmaceuticals

elexacaftor/tezacaftor/ivacaftor [Trikafta - US name]

treatment of cystic fibrosis November 4 2019medicine, orphan, accelerated assessment

not known pegfilgrastim reducing neutropenia due to chemotherapy October 3 2019 biosimilar

not known bevacizumab antineoplastic medicine October 3 2019 biosimlar

not known abiraterone treatment of prostate cancer October 3 2019 generic

not known lenalidomide immunosuppressant October 3 2019 generic

Novo Nordisk somapacitanpituitary and hypothalamic hormones and analogues

October 3 2019 medicine, orphan

Roche satralizumab

treatment of adult and adolescent patients with neuromyelitis optica spectrum disorder

October 3 2019medicine, orphan, accelerated assessment

not known rivaroxaban prevention of atherothrombotic events September 2 2019 generic

not known pegfilgrastim immunostimulant September 2 2019 biosimilar

not known fampridine treatment of multiple sclerosis September 2 2019 generic

not known dasatinib antineoplastic medicine September 2 2019 generic

not known budesonide/formoterol

treatment of asthma and chronic obstructive pulmonary disease

September 2 2019 generic

not known arsenic trioxidetreatment of relapsed acute promyelocytic leukemia

September 2 2019 generic

Gilead Sciences filgotinib treatment of adults with rheumatoid arthritis September 2 2019 medicine

Janssen rilpivirinefor use in combination with cabotegravir to treat HIV-1

September 2 2019 medicine

ViiV Healthcare cabotegravirfor use in combination with rilpivirine to treat HIV-1

September 2 2019 medicine

not known ioflupane (123I)

for detecting loss of functional dopaminergic neuron terminals in the striatum

August 7 2019 generic

Samsung Bioepis bevacizumab (SB8)

treatment of metastatic carcinoma of the colon or rectum, metastatic breast cancer and recurrence of platinum-sensitive epithelial ovarian, fallopian tube or primary peritoneal cancer

August 7 2019 biosimilar




Product Type/Status

Aimmune Therapeutics

arachis hypogaea allergens (AR101)

treatment of peanut allergy August 7 2019 medicine

Alnylam Pharmaceuticals givosiran

treatment of acute hepatic porphyria in adults and adolescents aged 12 years and older

August 7 2019medicine, orphan, accelerated assessment

Insmed amikacin (Arikayce)

treatment of lung infection as part of combination antibacterial drug regiment in adults

August 7 2019 medicine, orphan

Blueprint Medicines avapritinib

treatment of adult patients with PDGFRα D842V mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST

August 7 2019 medicine, orphan

Molecular Partners/Allergan abicipar pegol

treatment of neovascular age-related macular degeneration

August 7 2019 medicine

Sanofi isatuximabtreatment of patients with relapsed/refractory multiple myeloma

July 10 2019 medicine, orphan

Sanofi insulin aspart treatment of diabetes mellitus July 9 2019 biosimilar

not known trastuzumab antineoplastic medicine July 9 2019 biosimilar

not known doxorubicin antineoplastic medicine July 9 2019 generic

not known dasatinib treatment of leukemia July 9 2019 generic

not known apixaban prevention of venous thromboembolic events July 9 2019 generic

Elusys Therapeutics obiltoxaximabtreatment of inhalational anthrax due to Bacillus anthracis

July 9 2019 medicine, orphan

Novartis crizanlizumab treatment of sickle cell disease July 9 2019 medicine, orphan

Nabriva Therapeutics lefamulin

treatment of community-acquired pneumonia in adults 18 years of age and older

July 9 2019 medicine

Pacira BioSciences bupivacaine for postsurgical analgesia July 9 2019 medicine

Sign-up to a free Pink Sheet trial for access to the latest coverage.

https://pages.pharmaintelligence.informa.com/european-coverage-free-trial

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