From their meeting in July, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has published the highlights.
At this meeting there were sixteen recommendations for approvals of medicines, including two orphan medicines. A marketing authorisation was granted for Onpattro (patisiran), used for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy. Onpattro was reviewed under EMA’s accelerated assessment procedure, which is reserved for medicines of major public health interest. During its development Onpattro was designated as an orphan medicine.
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), which was designated as an orphan medicine during its development, received a positive opinion for the treatment of cystic fibrosis. Three biosimilar medicines also received a positive opinion from the Committee, which included: 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 Committee recommended extensions of therapeutic indication for the following twelve medicines: Abseamed, Binocrit, Blincyto, Darzalex, Epoetin alfa Hexal, Kalydeco, Mekinist, Nucala, Tafinlar, Xarelto and two extensions of indication for Keytruda.
Applications that were withdrawn include an initial marketing authorisation for Raligize (axalimogene filolisbac), which was intended to be used for the treatment of cervical cancer. Further applications that were withdrawn were the extension of the use of Opdivo (nivolumab) to the treatment of stomach cancer and Sutent (sunitinib) to treat patients at high risk of kidney cancer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.
For the meeting summary, and a full list of approvals, opinions and withdrawals, please click here.