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RSS FeedPosted by Michael Wonder on 03 Dec 2020
Aeglea BioTherapeutics receives FDA rare paediatric disease designation for ACN00177 for the treatment of homocystinuria
1 December 2020 - Company eligible to receive priority review voucher upon FDA approval of ACN00177. ...
Posted by Michael Wonder on 02 Dec 2020
BridgeBio Pharma and Affiliate QED Therapeutics announce FDA acceptance of new drug application for infigratinib for the treatment of cholangiocarcinoma
1 December 2020 - Application will also be submitted for review in Australia and Canada under Project Orbis. ...
Posted by Michael Wonder on 28 Nov 2020
Polaryx Therapeutics receives both rare paediatric disease and orphan drug designations for the treatment of GM2 gangliosidosis with PLX-300
25 November 2020 - Polaryx Therapeutics announced today that it has received from the U.S. FDA both rare paediatric disease and ...
Posted by Michael Wonder on 21 Nov 2020
ADC Therapeutics announces FDA accepts biologics license application and grants priority review for loncastuximab tesirine for treatment of relapsed or refractory diffuse large B-cell lymphoma
20 November 2020 - Prescription Drug User Fee Act target action date of 21 May 2021. ...
Posted by Michael Wonder on 18 Nov 2020
FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
18 November 2020 - Regulatory submission based on positive data from two trials in patients with late-onset and infantile-onset Pompe disease, ...
Posted by Michael Wonder on 11 Nov 2020
Angiocrine Bioscience announces FDA regenerative medicine advanced therapy designation granted to AB-205 (universal E-CEL cell therapy) to treat organ vascular niche injuries for the prevention of severe toxicities in lymphoma patients undergoing curative high-dose therapy with autologous stem cell transplantation
11 November 2020 - Angiocrine Bioscience today announced that the U.S. FDA granted the regenerative medicine advanced therapy designation for AB-205, ...
Posted by Michael Wonder on 09 Nov 2020
FDA grants rare paediatric disease designation to AMO Pharma for AMO-02 for treatment of congenital myotonic dystrophy
9 November 2020 - Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending ...
Posted by Michael Wonder on 03 Nov 2020
Gilead faces pressure to relinquish valuable FDA voucher awarded with remdesivir approval
2 November 2020 - A prominent advocacy group is asking Gilead Sciences to relinquish a valuable voucher that came with ...
Posted by Michael Wonder on 29 Oct 2020
FDA accepts for priority review Libtayo (cemiplimab-rwlc) for advanced non-small cell lung cancer with PD-L1 expression of ≥50%
29 October 2020 - Regeneron Pharmaceuticals today announced that the U.S. FDA has accepted for priority review the supplemental biologics license ...
Posted by Michael Wonder on 29 Oct 2020
reVision Therapeutics announces US FDA grant of rare paediatric disease and orphan drug designation for REV-0100 for the treatment of Stargardt disease
28 October 2020 - reVision Therapeutics today announced that the US FDA has granted the Company's request to designate REV-0100 as ...
Posted by Michael Wonder on 29 Oct 2020
Passage Bio’s PBKR03 receives orphan drug and rare paediatric disease designations from FDA for treatment of Krabbe disease
28 October 2020 - Passage Bio today announced that the U.S. FDA has granted orphan drug and rare paediatric disease designations ...
Posted by Michael Wonder on 28 Oct 2020
Enhertu granted priority review in the US for the treatment of HER2 positive metastatic gastric cancer
28 October 2020 - Only HER2 directed medicine to demonstrate significant improvement in overall survival compared to chemotherapy for previously ...
Posted by Michael Wonder on 28 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-104 to treat SURF1 associated Leigh syndrome
27 October 2020 - Taysha anticipated to submit Investigational new drug application for TSHA-104 to FDA in 2021. ...
Posted by Michael Wonder on 21 Oct 2020
Selecta Biosciences and AskBio receive FDA rare paediatric disease designation for their gene therapy for methylmalonic acidemia
20 October 2020 - Selecta Biosciences and Asklepios BioPharmaceutical today announced the U.S. FDA has granted rare paediatric disease designation to ...
Posted by Michael Wonder on 20 Oct 2020
Tagrisso granted priority review in the US for the adjuvant treatment of patients with early-stage EGFR-mutated lung cancer
20 October 2020 - Unprecedented results show treatment with Tagrisso reduced the risk of disease recurrence or death by 80% in ...