MYALEPTA (metreleptin) Receives Positive CHMP Opinion in Patients with Generalized and Partial Lipodystrophy

Novelion Therapeutics (NASDAQ:NVLN), a biopharmaceutical company dedicated to developing new standards of care for individuals living with rare diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of marketing authorization, under exceptional circumstances, for MYALEPTA.

As quoted in the press release:

The CHMP recommends granting marketing authorization for MYALEPTA, as an adjunct to diet, as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalized LD (Berardinelli-Seip syndrome); or acquired generalized LD (Lawrence syndrome) in adults and children 2 years of age and above; or with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.

The CHMP’s positive opinion will now be reviewed by the European Commission (EC), which has the authority to grant approval of this indication. The Company anticipates a decision in mid-2018.  If approved by the EC, MYALEPTA, which has orphan drug designation in Europe, will be the first and only licensed treatment in the EU as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in LD patients.

Click here to read the full press release.