X4 Pharmaceuticals receives rare paediatric disease designation from FDA for mavorixafor for the treatment of WHIM syndrome

10 December 2020 - X4 Pharmaceuticals today announced that it has received rare paediatric disease designation from the U.S. FDA ...

Read more →

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. ...

Read more →

Moleculin announces FDA approves 3 rare paediatric disease designations for WP1066

1 December 2020 - Moleculin Biotech today announced that the US FDA has approved its request for a rare paediatric ...

Read more →

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 ...

Read more →

PTC Therapeutics announces key regulatory designations for PTC596 to advance treatment of two rare oncology indications

18 November 2020 - PTC Therapeutics today announced that the United States FDA has granted PTC596 both orphan drug designation and ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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. ...

Read more →

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 ...

Read more →

Versantis receives FDA rare paediatric disease designation for VS-01 for the treatment of urea cycle disorders

19 October 2020 - Versantis today announced that the U.S. FDA has granted a rare paediatric disease designation to its lead ...

Read more →

Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome

14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis. ...

Read more →

Oxular receives rare paediatric disease and orphan drug designations for retinoblastoma treatment

12 October 2020 - Provides furthered momentum for OXU-003 development programme. ...

Read more →

Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis

9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020. ...

Read more →

Italfarmaco receives FDA rare paediatric disease designation for givinostat in Duchenne muscular dystrophy, announces completed enrolment in EPIDYS Phase 3 trial

9 October 2020 - The Italfarmaco Group provided today an update on the development of givinostat, its proprietary histone deacetylase ...

Read more →