The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has endorsed nine medicines for approval at its May meeting, including Novartis’ Aimovig, the first human monoclonal antibody for migraine prevention.
Aimovig (erenumab) belongs to a new class of medicines that work by blocking the activity of calcitonin gene-related peptide, a molecule that is involved in migraine attacks. Its mode of action differs from other similar migraine therapies in development, which target CGRP itself as opposed to the receptor pathway.
In clinical trials, after three months of treatment patients with chronic migraine showed a reduction of 2.5 monthly migraine days on average compared to placebo, while for patients with episodic migraine the reduction was either 1.3 or 1.8 days, depending on the dose taken.
Elsewhere, the Committee recommended granting a marketing authorisation for Ionis/Akcea Therapeutics’ Tegsedi (inotersen) for patients with hereditary transthyretin amyloidosis, a rare disease causing build up of amyloid the peripheral nervous system and multiple organs.
Tegsedi, an orphan medicine reviewed under the EMA’s accelerated assessment pathway, is an antisense oligonucleotide expected to decrease the production of transthyretin, which is defective in patients with the condition, to reduce the formation of amyloid.
The CHMP also issued a positive opinion for Aegerion Pharmaceuticals’ orphan drug Myalepta (metreleptin) for the treatment of leptin deficiency, a condition that causes severe obesity beginning in the first few months of life. The drug mimics the physiological effects of leptin by binding to and activating the human leptin receptor, thus decreasing various types of fat in the body and reducing their accumulation in tissues such as liver and muscle.
Otsuka Pharmaceutical Europe’s Rxulti (brexpiprazole) won backing as a treatment for schizophrenia. The antipsychotic binds primarily to dopamine D2 receptors, serotonin 5-HT1A and 5-HT2A receptors and noradrenergic α1B/2C receptors, to help improve symptoms of the condition.
Four biosimilar medicines received a positive opinion from the Committee: Sandoz’ Halimatoz, Hefiya and Hyrimoz, all biosimilars to AbbVie’s Humira (adalimumab), were recommended for the treatment of certain inflammatory and autoimmune disorders; and Pfizer’s Trazimera (trastuzumab), a biosimilar of Roche’s Herceptin, was backed for the treatment of breast and gastric cancer.
The Committee also supported approval of Cycle Pharmaceuticals’ generic medicine Nityr (nitisinone), for the treatment of hereditary tyrosinemia type 1, a genetic disorder characterised by elevated blood levels of the amino acid tyrosine, a building block of most proteins.
On the downside, a negative opinion was issued for Sarepta’s Exondys (eteplirsen) for the treatment of Duchenne muscular dystrophy.
Exondys 51 addresses the underlying cause of DMD by enabling the production of a functional dystrophin protein, and clinical studies have demonstrated a broadly favourable safety and tolerability profile as well as efficacy for the drug.
But the Committee said it was concerned that the main study, which involved just 12 patients, did not compare Exondys with placebo beyond 24 weeks, “during which there was no meaningful difference between Exondys and placebo in the 6-minute walking distance.”
The methods for comparing results of the main studies with historical data “were not satisfactory for showing that the medicine was effective,” it said, and noted that further data are needed “to show that the very low amounts of shortened dystrophin produced as a result of Exondys treatment bring lasting benefits relevant to the patient”.
Sarpeta has requested a re-examination of the decision.