1. Scrip
  2. >>Therapy Areas
  3. >>Musculoskeletal

Unsurprising FDA 'breakthrough' designation for Alexion's hypophosphatasia drug

 
• By Donna Young

Wall Street was not surprised on 28 May by the US FDA granting Alexion Pharmaceuticals' investigational hypophosphatasia (HPP) drug asfotase alfa (ENB-0040) a breakthrough therapy designation, given the high unmet medical need for the potentially life-threatening, ultra-rare metabolic genetic disease.

Wall Street was not surprised on 28 May by the US FDA granting Alexion Pharmaceuticals' investigational hypophosphatasia (HPP) drug asfotase alfa (ENB-0040) a breakthrough therapy designation, given the high unmet medical need for the potentially life-threatening, ultra-rare metabolic genetic disease.

After all, said Guggenheim Securities analyst Dr Bret Holley, there already was a high likelihood of US approval of asfotase alfa based on positive Phase II results, which

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 Musculoskeletal

Wave Aspires To Accelerated Approval In DMD With 48-Week Data

 
• By Joseph Haas

With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.

Regenxbio Rises After Duchenne Update And Sarepta Safety Scare

 
• By Andrew McConaghie

Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.

Sarepta Shares Plummet On News Of Elevidys Patient Death

 
• By Joseph Haas

The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.

Scrip Asks... What Does 2025 Hold For Biopharma? Part 6: Therapeutic Area Advances

 
• By Eleanor Malone

Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.

More from Therapeutic Category

Merck’s Welireg Adrenal Tumor Rollout Targets Specialized Centers

 
• By Alaric DeArment

The drugmaker won US FDA approval for the HIF-2α inhibitor in pheochromocytoma and paraganglioma, a rare cancer of the adrenal glands, in adolescents and adults.

Leo Roars To A Profit In Q1 As Adbry Grows In US

 
• By Kevin Grogan

The Danish firm's atopic dermatitis drug is holding its own despite strong competition from Sanofi/Regeneron’s Dupixent.

Multiple Launches Help Lilly’s Japan Growth

 
• By Ian Haydock

Lilly saw solid growth for its business in Japan last year on the back of several new launches and is building its obesity and Alzheimer's portfolios in the country.