FDA approves Zelboraf for Erdheim-Chester Disease6 Nov 2017
Zelboraf is the first FDA-approved treatment for Erdheim-Chester disease ECD, a rare blood disease.
Genentech, a member of the Roche Group, has announced that the FDA has approved Zelboraf (vemurafenib) for Erdheim-Chester disease (ECD) with BRAF V600 mutation. ECD is a rare, serious blood disease characterized by the abnormal multiplication of certain white blood cells called histiocytes, which can invade normal tissues and organs in the body.
The approval is based on data from the Phase II VE-BASKET study. Basket studies use an innovative clinical trial design that helps collect data faster and may accelerate the development of medicines for diseases with high unmet need. Instead of enrolling people based primarily on their disease or its location, basket studies match a disease’s underlying genetic profile to the mechanism of action of the medicine.
“This FDA decision means people living with Erdheim-Chester disease will now, for the first time, have an FDA-approved treatment option,” said Sandra Horning, chief medical officer and head of Global Product Development. “We are committed to finding new ways to bring medicines to patients with high unmet need, and we are pleased that this innovative clinical trial helped identify Zelboraf for treatment of this rare disease.”
“The Erdheim-Chester disease community is very encouraged by this first FDA-approved treatment for ECD, bringing new hope to patients and their families,” said Kathleen Brewer, president of ECD Global Alliance. “This new treatment option shows that meaningful breakthroughs can occur rapidly when patients, families, research physicians, industry and the FDA work together to help patients.”
Final results of VE-BASKET for the 22 people with ECD showed a best overall response rate of 54.5%. The most common Grade 3 or higher adverse events were new skin cancers, high blood pressure, rash and joint pain. The most common adverse events were joint pain, rash, hair loss, fatigue, change in heart rhythm and skin tags.
Zelboraf monotherapy was approved for the treatment of people with unresectable or metastatic melanoma with BRAF V600E mutation in 2011. The FDA previously granted Priority Review and Breakthrough Therapy Designation to Zelboraf for ECD with BRAF V600 mutation.
The 6-month FMD countdown - are you ready?
21 Aug 2018
With only 6 months left in which to become EU FMD-compliant, how can companies make the best use of their now limited options?Read more
Protecting the links between the UK and the EMA
14 Aug 2018
Now less than a year away from when the UK officially leaves the EU, the main goal remains the same - maintaining access to new drugs for patient health.Read more
TraceLink announces Russia compliance solution
13 Aug 2018
Company helps customers navigate and company with "one of the most complicated track and trace regulations in the world".Read more
A new era for migraine patients
6 Aug 2018
EU approves Novartis's Aimovig, a first-of-its-kind treatment specifically designed for migraine prevention.Read more
Breast cancer device receives FDA approval
3 Aug 2018
The first non-radioactive, dual-tracer for sentinel lymph node biopsy approved in the US.Read more
Building contingency plans for an ambiguous post-Brexit market
31 Jul 2018
One company's positive approach to Brexit preparations, despite the lack of definition.Read more
Post-Brexit medicines - stuck in the UK?
30 Jul 2018
The EMA needs to provide more action and guidance to help UK pharmaceutical manufacturers, says Thomas Beck, senior vice president, quality management, Recipharm.Read more
Pharmacovigilance post-Brexit - simpler than previously thought?
24 Jul 2018
To meet new PV regulations, the industry has to face a "multitude of unknowns and an ever-moving target".Read more
FDA approves first smallpox drug
17 Jul 2018
Action ensures that the US is prepared should smallpox be used as a bioweapon.Read more
TraceLink unveils Product Information Manager for its digital information sharing platform
12 Jul 2018
Product Information Manager addresses a broad range of use cases, beginning with saleable returns.Read more
Are you a supplier
Here's what we can do for you
- Generate quality leads for your business
- Stay visible for 365 days of the year
- Receive product inquiries and respond to meeting requests directly
- Improve company online presence through Search Engine Optimisation