The FDA has approved the drug Ayvakit developed by the biotechnology company Blueprint Medicines for the treatment of indolent systemic mastocytosis.
According to an article in BiopharmaDive, Ayvakit had been FDA-approved previously in 2021 based on a limited use for advanced mastocytosis. Adults are now covered to a greater extent for indolent (slow-moving) ISM. It is estimated that about 32,000 people in the United States are affected by the disorder and approximately ninety to ninety-five percent of these individuals have the ISM form.
About Systemic Mastocytosis
Systemic mastocytosis is a rare blood disorder that occurs when the body produces abnormal mast cells that multiply uncontrollably. These abnormal cells affect the skin and internal organs.
Mast cells are responsible for defending against intruders such as allergens. In systemic mastocytosis, abnormal mast cells set up a continuous allergic response. The cells also multiply uncontrollably, affecting your skin and internal organs. When the term ‘indolent’ is used it refers to a disorder that is slow growing.
About Getting to Market
Although Blueprint introduced Ayvakit and the cancer drug Gavreto recently neither drug has been a ‘big seller.’ ISM’s successful launch can help.
Currently, there are no medicines available that can alter the course of ISM. When tested, Ayvakit reduced the frequency of many unpredictable symptoms, namely brain fog.
The company recently conducted a conference call led by its CCO Philina Lee who told participants that it is confident Ayvakit will grow over time.
Blueprint executives told the conference call participants that the drug will be priced at a list price of about $445,000 per year which is the current price for the advanced disease.
The FDA approval was given in accordance with a study showing Ayvakit along with supportive treatment substantially mitigated symptoms when compared to a placebo plus additional care.
However, some analysts remain skeptical citing the drug’s price and other minor issues such as a new evaluation of efficacy. Andrew Berens, a securities analyst wrote that prescribing information appeared to erode efficacy from the level of marginal when comparing standard of care. He also commented on the newly included mention of hemorrhages and hematomas that had not been previously reported. The analyst noted that these issues may appear more often when subjected to longer exposure to the drug.
Future Competition
Note that Cogent Biosciences presents future competition with its ISM drug in a Phase 2 trial.