Kaerus Bioscience's lead candidate KER-0193 granted orphan drug designation and rare paediatric drug designation by US FDA for treatment of Fragile X syndrome

14 May 2025 - Kaerus Bioscience today announces that its lead candidate KER-0193 has been granted both orphan drug designation and ...

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FDA grants regenerative medicine advanced therapy designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable paediatric brain tumours

15 May 2025 - BrainChild Bio advancing BCB-276 towards BLA submission with a single pivotal Phase 2 trial planned to ...

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RegenxBio announces FDA acceptance and priority review of the BLA for RGX-121 for MPS II

13 May 2025 - FDA assigns PDUFA target action date of 9 November 2025. ...

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MeiraGTx granted FDA regenerative medicine advanced therapy designation for AAV-GAD for the treatment of Parkinson’s disease

9 May 2025 - This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD ...

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Health Canada accepts semaglutide 2.4 mg, a GLP-1 receptor agonist treatment for MASH, as a supplemental new drug submission under the priority review policy

2 May 2025 - Today, Novo Nordisk Canada announced that Health Canada has accepted for review, under the priority review policy, ...

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Innorna announces FDA rare paediatric disease designation granted to IN013 for treatment of Wilson disease

25 February 2025 - Innorna is thrilled to announce that the US FDA has granted rare paediatric disease designation to ...

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Fate Therapeutics receives regenerative medicine advanced therapy designation from FDA for FT819 to treat moderate to severe systemic lupus erythematosus

14 April 2025 - Fate Therapeutics today announced that the US FDA granted regenerative medicine advanced therapy designation to FT819, an ...

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Apellis announces FDA acceptance and priority review of the supplemental new drug application for Empaveli (pegcetacoplan) for C3G and primary IC-MPGN

1 April 2025 - PDUFA target action date is 28 July 2025. ...

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GRIN Therapeutics receives PRIME designation from EMA for radiprodil in the treatment of GRIN related neurodevelopmental disorder

1 April 2025 - GRIN Therapeutics today announced that the EMA has granted Priority Medicine (PRIME) designation to radiprodil, the company's ...

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Tolebrutinib regulatory submission accepted for priority review in the US for patients with multiple sclerosis

25 March 2025 - Tolebrutinib was granted breakthrough therapy designation by the FDA based on positive results from the HERCULES Phase ...

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US FDA grants priority review for new indication of finerenone for patients with common form of heart failure

17 March 2025 - Regulatory submission is based on positive results from the Phase 3 FINEARTS-HF study, which is part of ...

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Atsena Therapeutics granted US FDA fast track designation for ATSN-201 gene therapy to treat X-linked retinoschisis

12 March 2025 - Marks third FDA designation for ATSN-201, which has also received rare paediatric disease designation and orphan drug ...

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Capricor Therapeutics announces FDA acceptance and priority review of its biologics license application for deramiocel to treat Duchenne muscular dystrophy

4 March 2025 - FDA assigns PDUFA target action date of 31 August 2025. ...

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ImmunityBio receives FDA RMAT designation for Anktiva and CAR-NK for the reversal of lymphopaenia in patients receiving standard of care chemotherapy/radiotherapy and in treatment of multiply relapsed locally advanced or metastatic pancreatic cancer

27 February 2025 - Regenerative medicine advanced therapy designation follows clinical data of absolute lymphocyte count and significant overall survival correlations ...

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US FDA grants priority review to Sobi's supplemental biologics licence application for Gamifant (emapalumab-lzsg)

27 February 2025 - Sobi today announced that the US FDA has accepted the supplemental biologics license application for Gamifant (emapalumab-Izsg) ...

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