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Barry Cohen
Barry Cohen
Articles (195) 

Biomarin's Hemophilia A Drug Among 5 Up for FDA Approval Soon

If approved, company's Roctavian would become first gene therapy for inherited disease

Five pharmaceutical companies are awaiting approval of drugs in the next few months, and Biomarin Pharmaceutical Inc. (NASDAQ:BMRN) has the most to gain. If the company’s Roctavian gets greenlighted by the Food and Drug Administration, it will become the first gene therapy for one of the world’s highest-profile inherited diseases, hemophilia A.

Hemophilia is a rare hereditary bleeding disorder that occurs in a relatively small number of patients around the world. Its cause is a missing or deficient protein needed for blood clotting. Hemophilia A occurs in one in 5,000 live male births. It is about four times as common as hemophilia B.

The global hemophilia market is expected to reach more than $18 billion by 2027, according to a new report by Grand View Research Inc. It is anticipated to grow at a compound rate of 5.5% over the forecasted period.

Other public companies that compete in the hemophilia market are Pfizer Inc. (NYSE:PFE), Biotest (XTER:BIO), Novo Nordisk (NYSE:NVO), Bayer AG (BAYN), Roche (RHHBY), Grifols SA (XMAD:GRF) and Biogen Inc. (BIIB).

Seven patients who received the highest dose of Roctavian in a phase 1/2 study saw their bleeding episodes drop 95% over four years, according to an article in FierceBiotech. Before gene therapy, those patients needed more than 130 infusions of the clotting factor VIII to manage their disease each year; after treatment, that number dropped to an average of five infusions per year.

JPMorgan analyst Cory Kasimov called the data “Roc solid,” while SVB Leerink’s Joseph Schwartz said the treatment’s durability is “respectable.” The FDA is expected to make a decision on Roctavian by Aug. 21.

The other companies whose drugs the agency said they will make rule on are:

  • GlaxoSmithKline's (GSK) belantemab mafodotin: Glaxo appears to be leading the pack of about 12 companies vying to be the first to get the FDA OK for an antibody-cancer drug conjugate that targets a protein found on nearly all malignant cells. Other companies using the approach are Seattle Genetics Inc. (SGEN) and Roche. Those focusing on the protein using a different method are closing in on key milestones. They are Bristol-Myers Squibb Co. (BMY), bluebird bio Inc. (BLUE) and Regeneron Pharmaceuticals Inc. (REGN).
  • Nippon Shinyaku Co.'s (TSE:4516) viltolarsen: This medication is designed to treat the same Duchenne muscular dystrophy patients as Vyondys 53 from Sarepta Therapeutics Inc. (SRPT). Study results published in JAMA Neurology this past May suggest viltolarsen might even work better than the Sarepta drug, although comparing them might be misleading, reported BioPharma Dive
  • Gilead Sciences Inc.'s (GILD) filgotinib: This drug is the result of a research partnership between Gilead and Galapagos NV (GLPG). Filgotinib is in a drug class called JAK inhibitors. Its first target is rheumatoid arthritis, where the competition includes medications from Pfizer, Eli Lilly and Co. (LLY) and AbbVie Inc. (ABBV). Some Wall Street analysts think filgotinib might be safer than its rivals. The companies are banking on an August approval
  • Roche and PTC Therapeutics' (PTCT) risdiplam: The companies are anticipating approval in late August. If it gets the FDA’s OK, risdiplam will become the first treatment taken by mouth for spinal muscular atrophy, an often-fatal neuromuscular condition that primarily affects infants and children. The companies think risdiplam has advantages over two rival drugs, one from Biogen and Ionis Pharmaceuticals Inc. (IONS) and the other from Novartis (NVS).

Disclosure: The author has positions in Pfizer, Bristol-Myers Squibb, Gilead Sciences and Eli Lilly.

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About the author:

Barry Cohen
Barry Cohen has nearly 40 years experience in communications and marketing, the majority in senior positions at large international health care companies, including Abbott Laboratories and Bayer Inc.

He has contributed to a number of financial websites, writing primarily about the stocks of health care companies.

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