Among a host of positive recommendations announced Friday, EMA's CHMP backed approval of migraine therapy Aimovig erenumab and Orphan Drug designees Tegsedi inotersen for hereditary transthyretin (TTR) amyloidosis and Myalepta metreleptin for leptin deficiency.
The committee recommended approval of Aimovig, a mAb that inhibits the calcitonin gene-related peptide (CGRP) receptor, to prevent migraine. CHMP said it would be the first mAb approved in the indication; FDA approved Aimovig last month. Novartis AG (NYSE:NVS; SIX:NOVN) holds the drug's EU rights under a deal with Amgen Inc. (NASDAQ:AMGN).
CHMP backed approval of Tegsedi, a TTR antisense inhibitor from Akcea Therapeutics Inc. (NASDAQ:AKCA), which gained rights to the candidate in March from Ionis Pharmaceuticals Inc. (NASDAQ:IONS). EMA is reviewing it under accelerated assessment, and it is under Priority Review by FDA, with a July 6 PDUFA date; it has Orphan Drug designation in both jurisdictions.
Myalepta, a recombinant form of human leptin from Novelion Therapeutics Inc. (NASDAQ:NVLN), gained CHMP's recommendation to treat leptin deficiency in patients with generalized or partial lipodystrophy. FDA approved the drug in 2014 as Myalept.
The committee recommended approval of Rxulti brexpiprazole from Otsuka Pharmaceutical Co. Ltd. (Tokyo, Japan) and H. Lundbeck A/S (CSE:LUN) to treat schizophrenia in adults. FDA approved that therapy in 2015 as Rexulti. It is a partial agonist of the dopamine D2 receptor with affinity for multiple serotonin receptors.
Among four biosimilars whose approval CHMP recommended, three are biosimilars of Humira adalimumab from AbbVie Inc. (NYSE:ABBV). The Sandoz unit of Novartis is seeking approval of all three: Hyrimoz, Hefiya and Halimatoz. The fourth, Trazimera trastuzumab from Pfizer Inc. (NYSE:PFE), is a biosimilar of Herceptin from Roche (SIX:ROG; OTCQX:RHHBY).
As sponsor Sarepta Therapeutics Inc. (NASDAQ:SRPT) expected, CHMP declined to recommend Exondys eteplirsen, its therapy for Duchenne muscular dystrophy (DMD). Sarepta plans to request a re-examination. FDA granted accelerated approval to eteplirsen in 2016 as Exondys 51 to treat DMD patients amenable to exon 51 skipping. It is a phosphorodiamidate morpholino oligomer (PMO) that induces skipping of exon 51 in dystrophin mRNA.