Genentech’s Zelboraf® (vemurafenib) is a small molecule B-RAF inhibitor and is FDA-approved to treat patients with metastatic or unresectable melanoma whose tumors express the B-RAF V600E mutation. To reduce the likelihood of cancer cells becoming drug resistant, Genentech wanted to combine vemurafenib with cobimetinib, a small molecule MEK inhibitor that targeted a different part of … Continued
In 2020, the US FDA approved Risdiplam (Evrysdi®) as the first orally administered drug to treat SMA in patients ≥2 months old, followed by the European Medicine Agency. Risdiplam addresses the underlying cause of SMA: a reduced amount of survival motor neuron (SMN) protein.
Atypical hemolytic uremic syndrome (aHUS) is an ultra-rare genetic disease that causes abnormal blood clot formation in small blood vessels throughout the body leading to kidney failure, other organ damage, and premature death. At the start of this project, there were no FDA-approved aHUS treatments. Furthermore, as only a few thousand aHUS patients are diagnosed … Continued
Primary biliary cholangitis (PBC) is a chronic disease that impedes bile flow from the liver, resulting in increased bile acid concentration, which causes cell damage. Untreated PBC can lead to liver failure and death. At the start of this project, the only approved treatment for PBC was not effective for all patients.
A large biopharmaceutical company required insight on the current market access landscape in a rare disease, as well as feedback on a pipeline product that will launch as a second-to-market agent in the rare disease.
