Rare Blood Disease Drug Win Marks Fourth Approval for Blueprint Medicines

For the first time, patients facing an advanced form of rare hematologic disorder known as systemic mastocytosis (SM) have a targeted therapy aimed at the central driver of the disease.

On Wednesday, the U.S. Food and Drug Administration (FDA) approved Blueprint Medicines’ Ayvakit (avapritinib) for the treatment of adult patients with advanced SM, including aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) and mast cell leukemia (MCL). 

Advanced SM is a rare, hematologic disorder driven by the KIT D816V mutation in nearly all patients. The disease is characterized by uncontrolled mast cell proliferation and activation across multiple organ systems. That results in significant and often severe symptoms and extensive organ damage, causing a median overall survival of 3.5 years to less than six months.

Blueprint Chief Executive Officer Jeff Albers hailed the approval of Ayvakit as a needed therapy for the SM community. 

“As shown in two clinical trials, Ayvakit provides remarkable clinical efficacy to patients with advanced systemic mastocytosis, and this approval solidifies the therapy’s strong value proposition in this population.” Albers said in a statement. 

To Read the Complete Article at BioSpace, Click Here

Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

Stock image by @tatsianama from Depositphotos