DMD Approval, CAPR, Pig Kidney Transplant

Lickety-Split Analytics

Hello. This week we’re focused on Duchenne Muscular Dystrophy and the first pig to human kidney transplant. If you received this newsletter in your email, please send us a reply letting us know what you think.

-Ashley Adams

FDA Approves Duchenne Muscular Dystrophy Therapy

Last Thursday, the FDA approved Duvyzat (ginvinostat), an oral medication for the treatment of all genetic variants of Duchenne Muscular Dystrophy (DMD) in patients 6 years and older. It is the first nonsteroidal approval for DMD which is significant because it could:

1. Help mitigate negative side effects seen in steroidal options

2. Lead to better long-term efficacy, as tolerance might build up with steroid treatments

3. More directly slow disease progression compared to steroids that focus more on inflammation management

The company that developed this drug is a private company called Italfarmaco Group. DMD has seen a great breakthrough in development in recent years, with the first approval being granted to Sarepta Therapeutics in Sep 2016. Currently, there are 8 drugs on the market for DMD, many focused on different genetic mutation variants.

DMD is a rare, X-linked, genetic condition caused by mutations in the dystrophin gene. These mutations lead to improper levels of dystrophin, a protein that essentially helps protect muscles from damage during contraction and maintain muscle cell integrity. Experts estimate there are fewer than 50,000 people in the US with DMD, likely 15,000-20,000 people, with an occurrence rate of 1:3,500 male births worldwide. The disease is typically diagnosed in boys ages 3-6 exhibiting difficulty with everyday tasks like walking due to developing muscle weakness and atrophy, particularly in the pelvis area and shoulders. This may progress to being in a wheelchair by their teenage years and spread to loss of function in other areas of the body, ultimately leading to death through cardiomyopathy, breathing difficulties, and GI issues.

Other key upcoming catalyst for DMD development include:

• Sarepta (SRPT) in partnership with Roche (RHHBY) PDUFA date on 6/21/2024