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Roche-PTC Therapeutics’ oral spinal muscular atrophy…

By Shivani Singh

Aug 7 – The U.S. Food and Drug Administration on Friday approved an oral treatment from Roche Holding AG and PTC Therapeutics Inc for spinal muscular atrophy in adults and children two months and above.

The drug, risdiplam, to be sold as Evrysdi will compete with Biogen’s Spinraza and Novartis’ gene therapy, Zolgensma.

The list price of Evrysdi will be based on weight until the patient reaches 44 pounds (20 kg) at about 6 years of age, at which point the price reaches a maximum of $340,000 per year, Roche’s unit Genentech said in a statement.

The annual price of Evrysdi would be less than $100,000 for an infant who is less than 2 years old and weighs 15 pounds, a Genentech spokesperson said.

RBC Capital Markets analyst Brian Abrahams said the weight-based pricing comes as a slight surprise and is below the brokerage’s estimated $400,000 per year list price.

Evrysdi’s pricing compares with Biogen’s Spinraza, which lists at $750,000 for the first year and $375,000 annually thereafter, while Novartis’ Zolgensma, a one-time potentially curative therapy, lists for a whopping $2.1 million per patient.

Spinal muscular atrophy is the leading genetic cause of infant deaths that prevents the body from producing a protein necessary for neuromuscular development.

While neurologists see one-time therapy Zolgensma as likely the preferred treatment for many newborns with a severe form of the disease, some analysts see risdiplam’s once-daily oral regimen as a threat to Spinraza in older patients due to its more-intrusive administration process.

Evrysdi will be available in the United States within two weeks for direct delivery to patients’ homes through an Express Scripts specialty pharmacy.

Estimates of those living with the disease in the United States range from 10,000 to 25,000 people, according to the Spinal Muscular Atrophy Foundation.

PTC Therapeutics shares were up 3.7% after the bell. (Reporting by Manojna Maddipatla and Shivani Singh in Bengaluru and John Miller in Switzerland; Editing by Shailesh Kuber and Sriraj Kalluvila)

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