Santhera's drug gets US FDA nod for rare muscular dystrophy

[October 27, 2023]  (Reuters) -Catalyst Pharmaceuticals said on Thursday that U.S. health regulators have approved its partner Santhera Pharmaceuticals' drug to treat Duchenne muscular dystrophy (DMD) in patients aged two years and older.

Shares of Catalyst were up 3.8% in after market trading.

Swiss drugmaker Santhera had licensed the commercial rights for the manufacturing and supply of the drug, branded as Agamree, in North America to Catalyst for an upfront payment of $75 million and an equity investment of $15 million in the company.

The U.S. Food and Drug Administration's (FDA) approval comes just months after the approval of Sarepta Therapeutics' Elevidys, the first gene therapy for DMD.

DMD, a rare muscle-wasting disorder, is estimated to affect one in 3,500 male births worldwide

Santhera's drug works in a similar manner as corticosteroids like prednisone, which are the standard of care for Duschenne patients, but without the same safety concerns such as excessive weight gain and hair growth.

A spokesperson for Catalyst said the company has not released information about the drug's price or its specific commercial plans and will update towards the launch of the product in the United States, expected in the first quarter of 2024.

The company logo of Swiss-based Santhera Pharmaceuticals Holding AG is seen in front of its headquarters the Ceres Tower building in Pratteln, Switzerland July 18, 2019. REUTERS/Arnd WIegmann/File Photo

The regulatory approval for Agamree was supported by data from a pivotal mid-stage stage study where the drug showed comparable efficacy to current standard-of-care.

Following the commercial launch of the drug, Catalyst plans to introduce a financial assistance program to ensure accessibility and minimizing patient co-pays and deductibles.

The drug received the European regulator's backing earlier this month, and Santhera expects the European Commission's decision on authorization later this year.

(Reporting by Leroy Leo, Khushi Mandowara and Pratik Jain in Bengaluru; Editing by Maju Samuel and Shailesh Kuber)

[© 2023 Thomson Reuters. All rights reserved.]This material may not be published, broadcast, rewritten or redistributed.  Thompson Reuters is solely responsible for this content.