FDA Approves Orphan Drug Myalept - Pharmaceutical Technology

Latest Issue
PharmTech

Latest Issue
PharmTech Europe

FDA Approves Orphan Drug Myalept



AstraZeneca announced FDA approved orphan drug Myalept (metreleptin for injection), which is indicated as an adjunct to diet as replacement therapy for the treatment of complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. Myalept, a recombinant analogue of human leptin, is the first and only treatment approved by the FDA for these patients.

AstraZeneca is working to complete the transfer of the Biologics License Application for Myalept from Bristol-Myers Squibb to AstraZeneca as part of the acquisition of the diabetes alliance assets, including Myalept and Amylin Pharmaceuticals, which was completed on February 1, 2014.

The safety and effectiveness of Myalept for the treatment of complications of partial lipodystrophy or for the treatment of liver disease, including non-alcoholic steatohepatitis, have not been established. Myalept is not indicated for use in patients with HIV-related lipodystrophy or for use in patients with metabolic disease, including diabetes mellitus and hypertriglyceridaemia, without concurrent evidence of congenital or acquired generalized lipodystrophy.

Myalept is for subcutaneous injection only and is available in an 11.3 mg vial that requires reconstitution.

The FDA is requiring seven studies (post-marketing requirements) for Myalept, including a long-term prospective observational study (product exposure registry) of patients treated with Myalept, a study to assess for the immunogenicity (antibody formation) of Myalept, and an assessment and analysis of spontaneous reports of potential serious risks related to the use of Myalept. Eight additional studies are being requested as post-marketing commitments.

Source: AstraZeneca and FDA

ADVERTISEMENT

blog comments powered by Disqus
LCGC E-mail Newsletters

Subscribe: Click to learn more about the newsletter
| Weekly
| Monthly
|Monthly
| Weekly

Survey
FDASIA was signed into law two years ago. Where has the most progress been made in implementation?
Reducing drug shortages
Breakthrough designations
Protecting the supply chain
Expedited reviews of drug submissions
More stakeholder involvement
Reducing drug shortages
70%
Breakthrough designations
4%
Protecting the supply chain
17%
Expedited reviews of drug submissions
2%
More stakeholder involvement
7%
View Results
Eric Langerr Outsourcing Outlook Eric LangerTargeting Different Off-Shore Destinations
Cynthia Challener, PhD Ingredients Insider Cynthia ChallenerAsymmetric Synthesis Continues to Advance
Jill Wechsler Regulatory Watch Jill Wechsler Data Integrity Key to GMP Compliance
Sean Milmo European Regulatory WatchSean MilmoExtending the Scope of Pharmacovigilance Comes at a Price
From Generics to Supergenerics
CMOs and the Track-and-Trace Race: Are You Engaged Yet?
Ebola Outbreak Raises Ethical Issues
Better Comms Means a Fitter Future for Pharma, Part 2: Realizing the Benefits of Unified Communications
Better Comms Means a Fitter Future for Pharma, Part 1: Challenges and Changes

Click here