1. Scrip
  2. >>New Products

Sanofi's Xenpozyme Is First Drug Approved For Rare Genetic Disease ASMD

 
• By Jessica Merrill

The US FDA approved the enzyme replacement therapy for the condition also known as Niemann-Pick disease; it will be priced at a median annual cost of $780,000 per year.

Approval stamp
FDA approved the first drug for ASMD, a rare genetic disease. • Source: Shutterstock

The US Food and Drug Administration approved Sanofii's Xenpozyme (olipudase alfa) as the first treatment for non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) on 31 August. The ultra-rare genetic disease, historically known as Niemann-Pick disease types A, A/B and B, is a progressive disease linked to significant morbidity and mortality.

For Sanofi, the commercial opportunity will be relatively limited given the small patient numbers. Only 120 patients are diagnosed with ADMD in the US, two-thirds of whom are pediatric patients, according to Sanofi

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from New Products

Pipeline Watch: Thirteen Approvals And Ten Phase III Readouts

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

J&J’s Imaavy Approval Is The Starting Line For Pipeline-In-A-Product Strategy

 
• By Jessica Merrill

The company’s FcRn inhibitor nipocalimab was approved by the US FDA for generalized myasthenia gravis (gMG), where it will join a competitive market.

Pipeline Watch: Five Approvals And One Phase III Readout

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Merck KGaA Seals SpringWorks Buy

 
• By Kevin Grogan

The German group is paying $3.9bn to get hold of the US firm and its two approved products.

More from Scrip

Sarepta Slammed By Double Blow: Elevidys Sales Miss, Prasad’s CBER Appointment

 
• By Mandy Jackson

Sarepta’s stock price was battered after Vinay Prasad was appointed to lead the US FDA’s CBER, then the company’s shares were bruised when Q1 Elevidys sales came in below consensus.

Marea Eyes Cardioprotective Benefit With ANGPTL4 Inhibition

 
• By Joseph Haas

Phase IIa data for ANGPTL4-targeted antibody shows reduced triglycerides and remnant cholesterol, which Marea asserts indicates potential to protect against cardiovascular outcomes.

BeiGene To Trade Beach For Chalet In Swiss Redomiciling As BeOne

 
• By Alaric DeArment

The biotech saw a slight quarter-over-quarter decline in sales from the BTK inhibitor Brukinsa, which it attributed to seasonality, while revenues overall missed some analyst estimates.