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DMD Pipeline: After Sarepta’s First-Ever Approval, Are Combinations Next?

 
• By Mandy Jackson

Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.

DNA

Sarepta Therapeutics Inc.’s recent first-ever US FDA approval for a drug to treat Duchenne muscular dystrophy (DMD) is lending confidence for a wave of potential therapies and combination regimens to treat the rare disease – a small, but potentially lucrative market.

DMD is a devastating genetic disorder characterized by progressive muscle degeneration that affects one out of every 3,500 to 5,000 boys born worldwide. Over time patients lose the ability to...

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Lilly To Diversify Pain Pipeline With SiteOne Acquisition

 
• By Joseph Haas

Deal snapshot: With several internal non-opioid pain candidates in Phase II or earlier, Lilly looks to add R&D potential in pain with $1bn buyout of SiteOne.

Angelini Advances Brain Health Ambitions With GRIN Pact

 
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The Italian drugmaker gets rights to Phase III-ready asset radiprodil.

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The French drugmaker is expanding its neurology activities with the $470m deal.

In Brief: US FDA Delay Sends Biohaven Shares Down

 
• By Andrew McConaghie

The US regulator’s decision has taken Biohaven and investors by surprise, with the reason for a ‘major amendment’ status not clear.

More from Therapy Areas

Lilly To Diversify Pain Pipeline With SiteOne Acquisition

 
• By Joseph Haas

Deal snapshot: With several internal non-opioid pain candidates in Phase II or earlier, Lilly looks to add R&D potential in pain with $1bn buyout of SiteOne.

Angelini Advances Brain Health Ambitions With GRIN Pact

 
• By Kevin Grogan

The Italian drugmaker gets rights to Phase III-ready asset radiprodil.

GlycoEra’s Series B Cash Will Fund Efforts To Take On FcRn Inhibitor Class

 
• By Joseph Haas

GlycoEra unveiled a $130m series B financing, saying the proceeds would enable it to take its first clinical candidate through proof-of-concept in autoimmune diseases.