Sixteen medicines recommended for approval, including two orphans

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended 16 medicines for approval, including two orphan medicines, at its July 2018 meeting.

The Committee recommended granting a marketing authorisation for Onpattro (patisiran), for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (a condition in which the peripheral nerves are damaged). This medicine was reviewed under EMA’s accelerated assessment procedure, reserved for medicines of major public health interest. Onpattro was designated as an orphan medicine during its development.

The CHMP recommended granting two new paediatric-use marketing authorisations(PUMAs), for Kigabeq (vigabatrin), for the treatment of infantile spasms (West’s syndrome) and resistant partial epilepsy, and Slenyto (melatonin), for the treatment of insomnia in children and adolescents with autism spectrum disorder or Smith-Magenis syndrome.

Symkevi (tezacaftor / ivacaftor) received a positive opinion for the treatment of cystic fibrosis. Symkevi was designated as an orphan medicine during its development.

The Committee adopted a positive opinion for Xerava (eravacycline), a new antibacterial medicine for the treatment of complicated intra-abdominal infections in adults.

The CHMP recommended granting marketing authorisations for four cancer medicines: Braftovi (encorafenib) and Mektovi (binimetinib), two medicines to be used in combination in the treatment of patients with unresectable or metastatic melanoma with a BRAF V600 mutation; Imfinzi (durvalumab), for the treatment of non-small cell lung cancer and Verzenios (abemaciclib), for the treatment of locally advanced or metastatic breast cancer.

Ilumetri (tildrakizumab) received a positive opinion for the treatment of moderate to severe plaque psoriasis.

Three biosimilar medicines received a positive opinion from the Committee: Hulio(adalimumab), for the treatment of certain inflammatory and autoimmune disorders; Pelgraz (pegfilgrastim) and Udenyca (pegfilgrastim), both intended to reduce the duration of neutropenia and the incidence of febrile neutropenia due to chemotherapy.

The CHMP recommended granting a marketing authorisation for three generic medicinesDeferiprone Lipomed (deferiprone), for the treatment of iron overload in patients with thalassaemia major; Gefitinib Mylan (gefitinib), for the treatment of non-small cell lung cancer and Lenalidomide Accord (lenalidomide), for the treatment of multiple myeloma.

Negative recommendations on new medicines following re-examination

The applicants for Dexxience (betrixaban) and Eladynos (abaloparatide) requested re-examinations of the Committee’s negative opinions for these medicines adopted at the March 2018 meeting. After considering the grounds for these requests, the CHMPre-examined the initial opinions and confirmed its previous recommendations to refuse the granting of marketing authorisations for these medicines.

Twelve recommendations on extensions of therapeutic indication

The Committee recommended extensions of indication for Abseamed,  BinocritBlincyto,  Darzalex,  Epoetin alfa Hexal,  Kalydeco,  Mekinist,  Nucala,  Tafinlar,  Xarelto and two extensions of indication for Keytruda.

Negative opinions on extension of therapeutic indication

The CHMP adopted a negative opinion for the use of Opdivo (nivolumab) and Yervoy(ipilimumab) in combination to treat renal cell carcinoma (kidney cancer).

The Committee also adopted a negative opinion for an extension of therapeutic indication for Blincyto in patients with minimal residual disease after treatment for B-precursor acute lymphoblastic leukaemia.

Outcome of review on Xofigo

The CHMP recommended restricting the use of Xofigo (radium-223 dichloride) to patients who have had two previous treatments for metastatic prostate cancer or who cannot receive other treatments.

Withdrawals of applications

The application for an initial marketing authorisation for Raligize (axalimogene filolisbac) was withdrawn. This medicine was intended to be used for the treatment of cervical cancer.

Applications to extend the use of Opdivo (nivolumab) to the treatment of stomach cancer and Sutent (sunitinib) to treat patients at high risk of kidney cancer returning after surgery have also been withdrawn.

Posted on the EMA website on 27 July 2018