Blog
RSS FeedPosted by Michael Wonder on 22 Sep 2020
Medeor Therapeutics’ transplant immune tolerance therapy receives regenerative medicine advanced therapy designation from FDA
22 September 2020 - Medeor Therapeutics today announced that the U.S. FDA has granted a regenerative medicine advanced therapy designation ...
Posted by Michael Wonder on 21 Sep 2020
Crinetics Pharmaceuticals receives rare paediatric disease designation from FDA for CRN04777 for the treatment of congenital hyperinsulinism
21 September 2020 - Crinetics Pharmaceuticals today announced that the U.S. Food and Drug FDA has granted rare paediatric disease designation ...
Posted by Michael Wonder on 16 Sep 2020
FDA granted paediatric disease designation for OXi-4503
16 September 2020 - Treatment of acute myeloid leukaemia due to genetic mutations that disproportionately affect paediatric patients. ...
Posted by Michael Wonder on 15 Sep 2020
Ziopharm Oncology granted rare paediatric disease designation for controlled IL-12 for the treatment of DIPG
14 September 2020 - -- Ziopharm Oncology today announced that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 09 Sep 2020
Clarity Pharmaceuticals announces the US FDA grants rare paediatric disease designation to 64 Cu sartate, a diagnostic for the clinical management of neuroblastoma
9 September 2020 - Clarity Pharmaceuticals is pleased to announce that the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 31 Aug 2020
CorMedix announces FDA acceptance for filing and priority review of new drug application for Defencath
31 August 2020 - FDA sets PDUFA goal date of 28 February 2021. ...
Posted by Michael Wonder on 30 Aug 2020
FDA grants priority review of melflufen for patients with triple-class refractory multiple myeloma
29 August 2020 - Oncopeptides today announces that the US FDA has granted priority review for Oncopeptides' new drug application ...
Posted by Michael Wonder on 28 Aug 2020
Taysha Gene Therapies receives orphan drug designation and rare paediatric disease designation for TSHA-101 for GM2 gangliosidosis
27 August 2020 - TSHA-101 anticipated to advance into the clinic by the end of 2020. ...
Posted by Michael Wonder on 27 Aug 2020
Editas Medicine receives rare paediatric disease designation for EDIT-301 for the treatment of sickle cell disease
24 August 2020 - Editas Medicine today announced that the U.S. FDA has granted rare paediatric disease designation for EDIT-301, an ...
Posted by Michael Wonder on 26 Aug 2020
Sarepta Therapeutics announces FDA acceptance of casimersen (SRP-4045) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 45
25 August 2020 - Received FDA's conditional approval of Amondys 45 as brand name for casimersen. ...
Posted by Michael Wonder on 20 Aug 2020
U.S. FDA grants rare paediatric disease designation to AmideBio’s glucagon analog for the treatment of congenital hyperinsulinism
20 August 2020 - AmideBio announced today that the US FDA Offices of Pediatric Therapeutics and Orphan Products Development granted a ...
Posted by Michael Wonder on 18 Aug 2020
Adding sickle cell disease to FDA’s priority review voucher list of neglected tropical diseases
18 July 2020 - On 16 July, Putting Rare Diseases Patients First! filed a citizen’s petition to add sickle cell ...
Posted by Michael Wonder on 17 Aug 2020
G1 Therapeutics announces acceptance and priority review of NDA for trilaciclib for patients with small cell lung cancer
17 August 2020 - G1 launching expanded access program for patients with small cell lung cancer in the U.S.. ...
Posted by Michael Wonder on 12 Aug 2020
Theranexus and BBDF obtain orphan drug designation and rare paediatric disease designation from the FDA for BBDF-101 for Batten disease
11 August 2020 - Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation ...
Posted by Michael Wonder on 11 Aug 2020
Protalix BioTherapeutics and Chiesi Global Rare Diseases announce U.S. FDA acceptance of biologics license application for pegunigalsidase alfa for the proposed treatment of Fabry disease and grants priority review
11 August 2020 - Protalix BioTherapeutics together with its development and commercialization partner Chiesi Global Rare Diseases, a unit of Chiesi, ...