Blog
RSS FeedPosted by Michael Wonder on 11 Jun 2020
Rare disease therapy development and access remain top FDA priorities during COVID-19
11 June 2020 - The FDA’s work on behalf of people with rare diseases is more important than ever as these ...
Posted by Michael Wonder on 03 Jun 2020
Clarity Pharmaceuticals announces that the US FDA grants rare paediatric disease designation to Copper Cu 67 sartate for the treatment of neuroblastoma
3 June 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 26 Mar 2020
Gilead declines 'rare disease' status for experimental coronavirus drug
25 March 2020 - In a surprising turnabout, drugmaker Gilead Sciences asked the FDA on Wednesday to rescind orphan status ...
Posted by Michael Wonder on 26 Mar 2020
Orphan Technologies receives rare paediatric disease designation from FDA for OT-58 to treat cystathionine B-synthase deficiency homocystinuria
26 March 2020 - Orphan Technologies today announced that OT-58 for the treatment of cystathionine beta synthase deficiency homocystinuria has ...
Posted by Michael Wonder on 23 Feb 2020
Rare Disease Day 2020: FDA continues important work on treatments for rare diseases
21 February 2020 - As leaders of the FDA, every day we see the unwavering commitment of our agency in working ...
Posted by Michael Wonder on 16 Feb 2020
After a new version of a decades-old drug gets orphan status, the price suddenly skyrockets
12 February 2020 - For years, hospitals and clinics have used an injectable medicine called dehydrated alcohol to treat such ...
Posted by Michael Wonder on 30 Jan 2020
ProQR receives rare paediatric disease designation from FDA for QR-421a
30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...
Posted by Michael Wonder on 19 Jan 2020
FDA harnesses technology and collaboration to support rare disease product development
17 January 2020 - Announcing an orphan drug technology modernisation effort and the 2020 FDA Rare Disease Day meeting. ...
Posted by Michael Wonder on 27 Nov 2019
Our drug policy often prioritises eliminating rare disease. One researcher asks: Is that the right goal?
26 November 2019 - Dr. Peter Bach wants to turn the entire philosophical underpinnings of America’s health care system upside ...
Posted by Michael Wonder on 11 Nov 2019
FDA admits it goofed when granting orphan status to an opioid addiction treatment
8 November 2019 - In an unusual move, the FDA has acknowledged a mistake and revoked orphan drug status for ...
Posted by Michael Wonder on 08 Oct 2019
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases
8 October 2019 - The U.S. FDA today announced that it has awarded 12 new clinical trial research grants totaling ...
Posted by Michael Wonder on 03 Sep 2019
The FDA and Sarepta: a window into the real world of drug regulation
3 September 2019 - It is hard to discern the true state of drug regulation from the outside, but two ...
Posted by Michael Wonder on 20 Aug 2019
FDA accepts new drug application for VX-445 (elexacaftor), tezacaftor and ivacaftor combination treatment
20 August 2019 - FDA grants priority review of the application and sets a PDUFA target action date of 19 ...
Posted by Michael Wonder on 24 Jul 2019
Aeglea BioTherapeutics receives FDA breakthrough therapy designation for pegzilarginase for treatment of arginase 1 deficiency
24 July 2019 - Designation follows recently reported ARG1-D Phase 1/2 data demonstrating clinical response in patients rreated with pegzilarginase. ...
Posted by Michael Wonder on 22 Jul 2019
Vertex submits new drug application to the U.S. FDA for triple combination regimen of VX-445 (elexacaftor), tezacaftor and ivacaftor in cystic fibrosis
22 July 2019 - Application supported by positive results from two global Phase 3 studies in people with CF ages 12 ...