EMA’s human medicines committee (CHMP) recommended fifteen medicines for approval at its January 2020 meeting.
The Committee recommended granting a marketing authorisation for Givlaari* (givosiran), the first treatment for acute hepatic porphyria in adults and adolescents aged 12 years and older. Acute hepatic porphyria is a rare life-threatening genetic condition that causes attacks of severe abdominal pain, vomiting and nervous system disorders, such as seizures, depression and anxiety.
Givlaari benefited from support of the PRIME scheme, EMA’s platform for early and enhanced dialogue with developers of promising new medicines that address unmet medical needs. This interaction led to a more robust application package to demonstrate the medicine’s benefits and risks, which allowed its accelerated assessment. For more information, see the press release in the grid below.
The CHMP adopted a positive opinion for Rybelsus (semaglutide) for the treatment of adults with insufficiently controlled type 2 diabetes to improve glycaemic control as an adjunct to diet and exercise. It is the first glucagon-like peptide (GLP-1) receptor agonist treatment - a class of non-insulin medicines for people with type 2 diabetes - developed for oral use, providing patients with another option to treat the disease without injections. For more information, see the press release in the grid below.
The Committee recommended granting a marketing authorisation for Vaxchora (Cholera vaccine (recombinant, live, oral)) for prophylaxis against cholera, a very serious disease caused by Vibrio cholerae, in adults and children.
Liumjev (insulin lispro) received a positive opinion from the CHMP for the treatment of diabetes mellitus in adults.
The CHMP recommended granting marketing authorisations for Nilemdo (bempedoic acid) and Nustendi (bempedoic acid / ezetimibe) for the treatment of primary hypercholesterolaemia (high blood cholesterol that has no identifiable cause) and mixed dyslipidaemia (abnormally levels of fat in the blood).
Nubeqa (darolutamide) received a positive opinion for the treatment of prostate cancer.
The CHMP adopted a positive opinion for Staquis (crisaborole) for the treatment of atopic dermatitis.
The biosimilar medicine Ruxience (rituximab) received a positive opinion for the treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and Pemphigus vulgaris.
The CHMP recommended granting marketing authorisations for four generic medicines: Azacitidine betapharm (azacytidine) and Azacitidine Mylan (azacitidine), for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia; Arsenic trioxide Mylan (arsenic trioxide), for the treatment of acute promyelocytic leukaemia; and Cinacalcet Accordpharma (cinacalcet), for the treatment of secondary hyperparathyroidism, parathyroid carcinoma and primary hyperparathyroidism.
The CHMP recommended granting marketing authorisations for two hybrid medicines: Budesonide/Formoterol Teva Pharma B.V. (budesonide / formoterol fumarate dihydrate), for the treatment of asthma and chronic obstructive pulmonary disease; and Trepulmix* (treprostinil sodium), for the treatment of chronic thromboembolic pulmonary hypertension. Hybrid applications rely in part on the results of pre-clinical tests and clinical trials of an already authorised reference product and in part on new data.
The Committee recommended extensions of indication for Ameluz, MabThera, Rezolsta, Suliqua, Tybost and Venclyxto.
The application for an initial marketing authorisation for Idhifa (enasidenib) was withdrawn. This medicine was intended for the treatment of acute myeloid leukaemia, a cancer of white blood cells.
The application to extend the use of Keytruda (pembrolizumab) in the treatment of cancer of the oesophagus was also withdrawn.
Question-and-answer documents on these withdrawals are available in the grid below.
The agenda of the January meeting is published on EMA's website. Minutes of the December 2019 CHMP meeting will be published in the coming weeks.
Key figures from the January 2020 CHMP meeting are represented in the graphic below.
* This product was designated as an orphan medicine during its development. Orphan designations are reviewed by EMA's Committee for Orphan Medicinal Products (COMP) at the time of approval to determine whether the information available to date allows maintaining the medicine's orphan status and granting the medicine ten years of market exclusivity.
| Name of medicine | Givlaari |
| International non-proprietary name (INN) | givosiran |
| Marketing-authorisation applicant | Alnylam Netherlands B.V. |
| Therapeutic indication | Treatment of acute hepatic porphyria |
| More information | |
| Press release | First treatment for acute hepatic porphyria |
| Name of medicine | Liumjev |
| INN | insulin lispro |
| Marketing-authorisation applicant | Eli Lilly Nederland B.V. |
| Therapeutic indication | Treatment of diabetes mellitus in adults |
| More information | Liumjev: Pending EC decidion |
| Name of medicine | Nilemdo |
| INN | bempedoic acid |
| Marketing-authorisation applicant | FGK Representative Service GmbH |
| Therapeutic indication | Treatment of adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia |
| More information | Nilendo: Pending EC decision |
| Name of medicine | Nubeqa |
| INN | darolutamide |
| Marketing-authorisation applicant | Bayer AG |
| Therapeutic indication | Treatment of prostate cancer |
| More information | Nubega: Pending EC decision |
| Name of medicine | Nustendi |
| INN | bempedoic acid / ezetimibe |
| Marketing-authorisation applicant | FGK Representative Service GmbH |
| Therapeutic indication | Treatment of primary hypercholesterolaemia and mixed dyslipidaemia |
| More information | Nustendi: Pending EC decision |
| Name of medicine | Rybelsus |
| INN | semaglutide |
| Marketing-authorisation applicant | Novo Nordisk A/S |
| Therapeutic indication | Treatment of type 2 diabetes |
| More information | Rybelsus: Pending EC decision |
| Press release | First oral GLP-1 treatment for type 2 diabetes |
| Name of medicine | Staquis |
| INN | crisaborole |
| Marketing-authorisation applicant | Pfizer Europe MA EEIG |
| Therapeutic indication | Treatment of atopic dermatitis |
| More information | Staquis: Pending EC decision |
| Name of medicine | Vaxchora |
| Common name | Cholera vaccine (recombinant, live, oral) |
| Marketing-authorisation applicant | Emergent Netherlands B.V. |
| Therapeutic indication | Prophylaxis against disease caused by Vibrio cholerae in adults and children |
| More information | Vaxchora: Pending EC |
| Name of medicine | Ruxience |
| INN | rituximab |
| Marketing-authorisation applicant | Pfizer Europe MA EEIG |
| Therapeutic indication | Treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukaemia, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis, and Pemphigus vulgaris |
| More information | Ruxience: Pending EC decision |
| Name of medicine | Arsenic trioxide Mylan |
| INN | arsenic trioxide |
| Marketing-authorisation applicant | Mylan Ireland Limited |
| Therapeutic indication | Treatment of acute promyelocytic leukaemia |
| More information | Arsenic trioxide Mylan: Pending EC decision |
| Name of medicine | Azacitidine betapharm |
| INN | azacitidine |
| Marketing-authorisation applicant | betapharm Arzneimittel GmbH |
| Therapeutic indication | Treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia |
| More information | Azacitidine betapharm: Pending EC decision |
| Name of medicine | Azacitidine Mylan |
| INN | azacitidine |
| Marketing-authorisation applicant | Mylan Ireland Limited |
| Therapeutic indication | Treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia |
| More information | Azacitidine Mylan: Pending EC decision |
| Name of medicine | Cinacalcet Accordpharma |
| INN | cinacalcet |
| Marketing-authorisation applicant | Accord Healthcare S.L.U. |
| Therapeutic indication | Treatment of secondary hyperparathyroidism, parathyroid carcinoma and primary hyperparathyroidism |
| More information | Cinacalcet Accordpharma: Pending EC decision |
| Name of medicine | Budesonide/Formoterol Teva Pharma B.V. |
| INN | budesonide / formoterol fumarate dihydrate |
| Marketing-authorisation applicant | Teva Pharma B.V. |
| Therapeutic indication | Treatment of asthma and chronic obstructive pulmonary disease |
| More information | Budesonide / Formoterol Teva Pharma B.V.: Pending EC decision |
| Name of medicine | Trepulmix |
| INN | treprostinil sodium |
| Marketing-authorisation applicant | SciPharm Sarl |
| Therapeutic indication | Treatment of chronic thromboembolic pulmonary hypertension |
| More information | Trepulmix: Pending EC decision |
| Name of medicine | Ameluz |
| INN | 5-aminolevulinic acid |
| Marketing-authorisation holder | Biofrontera Bioscience GmbH |
| More information | Ameluz: Pending EC decision |
| Name of medicine | MabThera |
| INN | rituximab |
| Marketing-authorisation holder | Roche Registration GmbH |
| More information | MabThera: Pending EC decision |
| Name of medicine | Rezolsta |
| INN | darunavir / cobicistat |
| Marketing-authorisation holder | Janssen-Cilag International NV |
| More information | Rezolsta: Pending EC decision |
| Name of medicine | Suliqua |
| INN | insulin glargine / lixisenatide |
| Marketing-authorisation holder | sanofi-aventis groupe |
| More information | Suliqua: Pending EC decision |
| Name of medicine | Tybost |
| INN | cobicistat |
| Marketing-authorisation holder | Gilead Sciences Ireland UC |
| More information | Tybost: Pending EC decision |
| Name of medicine | Venclyxto |
| INN | venetoclax |
| Marketing-authorisation holder | AbbVie Deutschland GmbH & Co. KG |
| More information | Venclyxto: Pending EC decision |
| Name of medicine | Idhifa |
| INN | enasidenib |
| Marketing-authorisation holder | Celgene Europe B.V. |
| More information | Idhifa: Withdrawn application |
| Name of medicine | Keytruda |
| INN | pembrolizumab |
| Marketing-authorisation holder | Merck Sharp & Dohme B.V. |
| More information | Keytruda: Withdrawn application |