Novartis Receives Third FDA Breakthrough Designation - Pharmaceutical Technology

Latest Issue
PharmTech

Latest Issue
PharmTech Europe

Novartis Receives Third FDA Breakthrough Designation


The US Food and Drug Administration (FDA) has granted Novartis breakthrough therapy designation—the company’s third such designation this year—to BYM338 for sporadic inclusion body myositis (sIBM).

FDA crated the breakthrough therapy designation to expedite the development and review of new drugs for serious or life-threatening conditions. The designation requires preliminary clinical evidence that demonstrates substantial improvement over currently available therapy. The designation includes all of the fast track program features, as well as more intensive FDA interaction and guidance.

Novartis reports that the breakthrough designation is based on the results of a Phase II proof-of-concept study that showed BYM338 substantially benefited patients with sIBM compared to placebo. The results of this study will be presented at the American Neurological Association meeting on Oct. 14 and is expected to be published in a major medical journal later this year, according to a company statement.

sIBM is a rare, yet potentially life-threatening muscle-wasting condition. Patients who have the disease can gradually lose the ability to walk, experience falls and injuries, lose hand function, and have swallowing difficulties.

"BYM338 is the third example this year of Novartis' leadership in bringing breakthrough therapies to patients reinforcing our commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients," said Timothy Wright, M.D., Global Head of Development, Novartis Pharmaceuticals. "With no effective therapies currently available for sIBM, bimagrumab has the potential to be the first real option for patients with this condition."

BYM338 (bimagrumab) is a novel, fully human monoclonal antibody developed to treat pathological muscle loss and weakness. BYM338 was developed by the Novartis Institutes for Biomedical Research, in collaboration with Morphosys, whose HuCAL library was used to identify the antibody. BYM338 binds with high affinity to type II activin receptors, preventing natural ligands from binding, including myostatin and activin. BYM338 stimulates muscle growth by blocking signaling from these inhibitory molecules.

Earlier this year, Novartis received Breakthrough Therapy designation status for RLX030 (serelaxin), an investigational treatment for patients with acute heart failure (AHF) and LDK378 for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer.

 

Source: Novartis

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