1. Scrip
  2. >>Rare Diseases

Marinus, With Ztalmy Approval, Takes First Step Into Seizure Disorder Market

 
• By Jessica Merrill

The company will launch Ztalmy for an ultra-rare pediatric seizure disorder but hopes to expand to larger indications in the future. CEO Scott Braunstein talks to Scrip about the launch.

start line
Marinus has reached its commercial start • Source: Alamy

Marinus Pharmaceuticals, Inc. has secured its first US Food and Drug Administration approval, and it is for Ztalmy (ganaxolone) for a rare pediatric seizure disorder, coming after a long development road and several failed clinical trials. The company announced the FDA approval of Ztalmy, an oral suspension, on 18 March for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDD) in patients two years of age and older.

The initial approval is for an ultra-rare pediatric disorder that only affects about 75 to 100 newborns each year in...

More from Rare Diseases

More from Scrip

China Signals Restarting IPOs For Unprofitable Biotechs

 
• By Dexter Jie Yan

The China Securities Regulatory Commission has proposed a new “growth tier” for the STAR Market of the Shanghai Stock Exchange, to reinstate the market’s listing standards for unprofitable firms.

BIO Notebook: Woodcock Calls For Doing The Right Thing, Dealmaking Remains Constrained

 
• By Mandy Jackson, Joseph Haas, and Sarah Karlin-Smith

Highlights from Day Four of the BIO International Convention include Woodcock offering practical advice on rare disease trials, the sorry state of dealmaking mid-year, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

Syncona Looks To Go Private As Market Decline Deepens

 
• By Andrew McConaghie

While some are calling for Syncona to be wound up, the UK-based company believes many existing and new investors will back the creation of new private fund.