The U.S. Food and Drug Administration has approved Sanofi’s (SASY.PA), opens new tab drug to treat a type of blood disorder, the company said on Friday, in a sign that the French drugmaker’s $3.7 billion bet on the treatment could start to pay off.
The approval aids the company’s broader push to become a power house of rare disease and immunology drugs, as it looks to drive growth beyond its top-selling eczema treatment, Dupixent, on which it partners with Regeneron Pharmaceuticals (REGN.O), opens new tab.
Sanofi received access to the oral drug, rilzabrutinib, through its deal for Principia Biopharma in 2020. Leerink analysts expect the treatment to bring in peak sales of $2 billion to $5 billion.
Rilzabrutinib, to be sold under the brand name Wayrilz, received approval for use in adult patients with the condition known as persistent or chronic immune thrombocytopenia (ITP) who have stopped responding to prior treatments.
Sanofi plans to make the drug available to U.S. patients in September. The drugmaker has set a list price of $17,500 for a month’s supply of the treatment, but actual costs for patients will vary based on their insurance plans.
The condition is a rare blood disorder in which the immune system mistakenly attacks and destroys platelets, which help blood clot after an injury.
Patients with such condition often have dangerously low platelet levels — typically below 100,000 per microlitre, compared to the normal range of 150,000 to 400,000 — raising the risk of serious bleeding, including potentially fatal brain hemorrhages. Other common symptoms of the condition include easy bruising, fatigue and muscle aches.
“I think the big-ticket item here is not just the platelet count, it’s how patients feel, which is the biggest problem with ITP, where between 40% and 60% of patients really feel helpless,” Houman Ashrafian, Sanofi’s head of R&D, told Reuters ahead of the decision.
Wayrilz works by targeting Bruton’s tyrosine kinase (BTK), a protein involved in the communication of immune cells, potentially leading to a reduction in the overactive immune responses.
