As I mentioned in my September post about Bosulif, CML patients have a specific type of genetic mutation called the Philadelphia chromosome. This mutation causes bone marrow to make an enzyme that triggers the abnormal growth of white blood cells.
Synribo also goes knocks out the protein signal responsible for cancer cell growth. It is administered via daily under-the-skin injections in 28-day cycles. The first cycle consists of injections twice a day for 14 days. The second and subsequent cycles are twice a day for seven days and last as long as the patient continues to respond to treatment.
Synribo is approved under the FDA’s accelerated approval program. This program gives patients earlier access to promising new drugs on the condition that the drug meets a surrogate endpoint (think specific benchmark) that indicates how it will work in patients and undergoes additional studies that confirm its benefit.
But even when accelerated, the process takes a long time. Which is why we are advoating to get new therapies to patients faster, bridging the funding gap for research and drugs with strong promise and paving the way via venture philanthropy with pharmaceutical companies.