{"id":29740,"date":"2023-06-30T14:31:37","date_gmt":"2023-06-30T18:31:37","guid":{"rendered":"https:\/\/ifintechworld.com\/uncategorized\/us-fda-approves-biomarins-gene-therapy-for-hemophilia-a\/"},"modified":"2023-06-30T14:31:38","modified_gmt":"2023-06-30T18:31:38","slug":"us-fda-approves-biomarins-gene-therapy-for-hemophilia-a","status":"publish","type":"post","link":"https:\/\/ifintechworld.com\/?p=29740","title":{"rendered":"US FDA approves BioMarin’s gene therapy for hemophilia A"},"content":{"rendered":"
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\u00a9 Reuters. FILE PHOTO: The corporate logo of the U.S. Food and Drug Administration (FDA) is shown in Silver Spring, Maryland, November 4, 2009. REUTERS\/Jason Reed\/File Photo<\/span>
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By Nathan Gomes<\/p>\n

(Reuters) -The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical (NASDAQ:)’s gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding disorder an alternative to regular injections of missing blood proteins.<\/p>\n

It priced the one-time therapy, Roctavian, at $2.9 million. Pivotal trial results showed that Roctavian reduced bleeding events, but its durability is not known, and the company said it would include a warranty to health insurers. <\/p>\n

BioMarin said most study participants continued to respond to the gene therapy through year three and beyond, and it would continue to monitor them for 15 years.<\/p>\n

Gene therapies use a deactivated virus to deliver the needed genes and the immune system would recognize and eliminate a second dose of the therapy. <\/p>\n

The company said it would offer all U.S. health insurers a “warranty”, under which it would reimburse the wholesale cost if a patient does not respond. The company said the reimbursement would be “pro-rated” for patients that stopped responding in the first four years after treatment.<\/p>\n

Roctavian is the first gene replacement therapy for the most common form of hemophilia, enabling patients a way to forego or reduce the need for lifetime treatment with factor proteins needed to help blood clot or with monthly doses of a newer antibody drug. <\/p>\n

Shares of the California-based company closed 3.6% lower on Thursday.<\/p>\n

The approval was pretty widely expected and now there is debate on how the uptake will pan out, said Joel Beatty, senior research analyst at Robert W Baird.<\/p>\n

Roctavian, which is manufactured at the company’s facility in Novato, California, works by delivering a functional copy of the missing gene that would help hemophilia A patients make a blood-clotting protein known as factor VIII.<\/p>\n

“Roctavian’s launch will probably be a little slow at first, because there are already some effective treatment options on the market. But longer-term, one-time dosing will likely end up being attractive to a lot of patients,” Beatty added.<\/p>\n

BioMarin’s therapy will compete for market share with Roche’s Hemlibra, an antibody drug which mimics the function of the blood clotting protein missing in hemophilia A patients.<\/p>\n

Roctavian was approved for hemophilia A in the European Union last year, and is sold under the same brand name.<\/p>\n

In April, BioMarin cut its annual sales forecast range for Roctavian to $50 million to $100 million, from $100 million to $200 million.<\/p>\n

The company said on Thursday it expects about 2,500 of around 6,500 adults living with severe hemophilia A in the United States to be eligible to receive Roctavian with this initial approval.<\/p>\n

There are about 16,000 patients in the United States with hemophilia A in which they are missing the factor VIII clotting protein.<\/p>\n<\/div>\n

Read the full article here<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

\u00a9 Reuters. FILE PHOTO: The corporate logo of the U.S. Food and Drug Administration (FDA) is shown in Silver Spring, Maryland, November 4, 2009. REUTERS\/Jason Reed\/File Photo By Nathan Gomes (Reuters) -The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical (NASDAQ:)’s gene therapy for severe hemophilia A, the company said, giving patients with […]<\/p>\n","protected":false},"author":1,"featured_media":6537,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"video","meta":{"footnotes":""},"categories":[1],"tags":[83],"class_list":["post-29740","post","type-post","status-publish","format-video","has-post-thumbnail","hentry","category-uncategorized","tag-featured","post_format-post-format-video"],"yoast_head":"\nUS FDA approves BioMarin's gene therapy for hemophilia A | iFintechWorld<\/title>\n<meta name=\"description\" content=\"\u00a9 Reuters. 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