The deal will bolster nthye French company’s drug development portfolio with an experimental treatment for a rare genetic disease.
Tag Archive for: rare disease
Approaching the start of their 7th year in business as a full-service healthcare agency, QBFox Healthcomm is taking on the new year with renewed resolve. Driven by new business, a key promotion, and expanding staff, the agency continues on its roll.
A legislative proposal, if passed into law, could cost the United States up to 1.2 million jobs and lead to more than 130 fewer FDA approvals over a 10-year period.
Iovance’s lifileucel for advanced melanoma and Madrigal’s resmetirom for nonalcoholic steatohepatitis are just a couple of the key decisions on the FDA’s docket this quarter.
The drug, branded as Wainua, is approved for patients with polyneuropathy, or nerve damage caused by hereditary transthyretin amyloidosis (ATTR-PN), which affects an estimated 40,000 patients globally.
In the next two weeks, the agency will release regulatory decisions on investigational treatments for a rare hereditary disorder, non-small cell lung cancer and more.
Ahead of an expected surge in regulatory filings, the FDA is establishing the Genetic Metabolic Diseases Advisory Committee to provide advice on treatments for these complex and challenging conditions.
The Swiss pharma’s Fabhalta, a Factor B inhibitor, is the first FDA-approved oral monotherapy for adults with the rare, chronic blood disorder paroxysmal nocturnal hemoglobinuria.
With the biopharma industry’s looming wave of gene therapy submissions and potential approvals, the senior senator is laying the groundwork for a legislative initiative to improve access to these expensive treatments.
Centron and Fusion Medical Communications are uniting under one agency as Propel Health Communications.
