Rilzabrutinib

Bolstering Sanofi's immunology and inflammation (I&I) growth aspirations, the FDA on Friday approved the French pharma's Wayrilz (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The nod lends credence to Sanofi's $3.7-billion purchase of Principia Biopharma to gain the asset in 2020, as Wayrilz now represents a potential key growth driver for its I&I pipeline — an area the drugmaker is increasingly relying on for revenue as it slims down its oncology portfolio. For related analysis, see Vital Signs: Sanofi builds momentum for its I&I bets.An oral BTK inhibitor, Sanofi said Wayrilz can get at the root causes of ITP by targeting different pathways across the immune system."With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy," Brian Foard, Sanofi's head of specialty care, said in a company release. "Its multi-immune modulation approach shows promise in addressing the key drivers of immune thrombocytopenia, which aligns with Sanofi's commitment to adapting and evolving therapeutic solutions to help tackle ongoing unmet patient needs."Durable platelet responseThe approval was based on results from the Phase III LUNA 3 study, which were toplined last April before being shared in more detail at last year's American Society of Hematology (ASH) meeting. The 202-patient study compared twice-daily oral Wayrilz against placebo. The BTK inhibitor met the trial's primary endpoint of durable platelet response, with 23% of patients who received the therapy achieving platelet counts at or above 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, compared with zero patients in the placebo group. Additionally, Wayrilz reduced the need for rescue therapy by a significant 52% versus placebo, and also led to significant and clinically meaningful improvements in physical fatigue and bleeding. The most common treatment-related adverse events were diarrhoea (23%), nausea (17%), headache (8%) and abdominal pain (6%).Feedback from experts suggest Wayrilz is likely to become a go-to second-line option after thrombopoietin receptor agonists, with the potential to quickly gain traction due to physician familiarity with BTK inhibitors such as Imbruvica (ibrutinib) in blood cancers. However, Sanofi has plenty of competition nipping at its heels from Novartis' BAFF-R antibody ianalumab and several anti-CD38 mAbs. For further analysis, see KOL Views Q&A: Sanofi's rilzabrutinib can contend in ITP but competition fierce and more footsteps approaching.Outside the US, Wayrilz is under regulatory review in the EU and China.

Bengaluru: The U.S. Food and Drug Administration has approved Sanofi's 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. 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. (Reporting by Bhanvi Satija, Puyaan Singh and Sriparna Roy in Bengaluru; Editing by Shilpi Majumdar and Maju Samuel)