FDA Approves Amgen’s Soliris Biosimilar & More — This Week in RARE Daily

May 31, 2024

This Week in RARE Daily is a feature from Global Genes where you can get a quick rundown of the top 5 headlines in the rare disease space from our editorial staff. Here are top stories from this past week, May 24 – 30, 2024:

FDA Approves Amgen’s Soliris Biosimilar

The U.S. Food and Drug Administration approved Amgen’s Bkemv as the first interchangeable biosimilar to Soliris to treat paroxysmal nocturnal hemoglobinuria patients with atypical hemolytic uremic syndrome.
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Asahi Kasei Offers to Buy Calliditas for $1.1 Billion
Japanese pharma Asahi Kasei is expanding its rare disease business with an offer to acquire Swedish biotech Calliditas Therapeutics for $1.1 billion in cash, a premium of 83 percent to the closing price of its Swedish shares and 74 percent to the closing price of its ADS, prior to the offer.
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LifeArc and ALS Therapy Development Institute Collaborate to Find and Validate Biomarkers

The UK charity LifeArc and the US non-profit ALS Therapy Development Institute said they are collaborating to identify, validate, and develop new biomarkers that are needed to improve the evaluation of patients and drug development for the rare neurodegenerative disease amyotrophic lateral sclerosis.
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Day One Sells Priority Review Voucher for $108 Million
Day One Biopharmaceuticals said it sold its Priority Review Voucher for $108 million to an undisclosed buyer. The company was awarded the Priority Review Voucher following the FDA’s accelerated approval of Ojemda.
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NS Pharma’s Viltepso Fails Confirmatory Trial in Duchenne Muscular Dystrophy

NS Pharma, a subsidiary of Nippon Shinyaku, said preliminary analysis showed its Duchenne muscular dystrophy drug Viltepso failed the primary endpoint in a global late-stage trial in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.
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