5. FDA Approves First Therapy for Rare, Debilitating Tumor

Pexidartinib (Turalio, Daiichi Sankyo) is indicated for adult patients with symptomatic tenosynovial giant cell tumor associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Read more.

4. Cancer Cell Growth Mechanism Discovered

Research could lead to the development of new cancer treatments to stop tumor growth. Read more.

3. The Journey of Commitment to Ultra-Rare Disease Patients: A Supportive Business Model

Over the last 5 years, more than 4000 FDA-regulated clinical trials were initiated worldwide for rare disease treatments. Read more.

2. Drug Combination Leads to 4-Year Survival in Patients with Lung Cancer

A regimen of afatinib as a first-line treatment followed by osimertinib in patients with epidermal growth factor receptor mutation-positive non-small cell lung cancer was found to significantly improve overall survival. Read more.
 

1. FDA Monitoring Data Accuracy Issues with Gene Therapy Product

Officials with the FDA are assessing data accuracy issues related to Zolgensma, but have no major concerns about the spinal muscular atrophy treatment remaining on the market. Read more.