CRISPR Therapeutics announces FDA regenerative medicine advanced therapy designation granted to CTX130 for the treatment of cutaneous T-cell lymphomas

28 September 2022 - CRISPR Therapeutics today announced that the US FDA granted regenerative medicine advanced therapy designation to CTX130, ...

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Pharming announces US FDA acceptance for priority review of its new drug application for leniolisib

28 September 2022 - The FDA has assigned a PDUFA goal date of 29 March 2023 for the NDA submission based ...

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FDA grants rare paediatric disease designation to dovitinib for osteosarcoma

22 September 2022 - Rare paediatric disease designation qualifies Oncoheroes to receive fast track review, and a priority review voucher at ...

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Cidara Therapeutics announces FDA acceptance for priority review of new drug application for rezafungin for the treatment of candidemia and invasive candidiasis

20 September 2022 - Assigned PDUFA target action date of 22 March 2023. ...

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AvroBio receives rare paediatric disease designation from US FDA for first gene therapy in development for cystinosis

20 September 2022 - AVR-RD-04 has previously received orphan drug designation from FDA and EMA. ...

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Seagen announces Tukysa (tucatinib) in combination with trastuzumab granted priority review by FDA for previously treated HER2 positive metastatic colorectal cancer

19 September 2022 - FDA has set action date of 19 January 2023 . ...

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FDA grants Radiopharm Theranostics DUNP19 product rare paediatric disease designation

16 September 2022 - Radiopharm Theranostics has been granted rare paediatric disease designation by the US FDA for its DUNP19 ...

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Aro Biotherapeutics receives FDA rare paediatric drug designation for ABX1100 for the treatment of Pompe disease

15 September 2022 - Aro Biotherapeutics today announced that the US FDA granted rare paediatric drug designation for ABX1100, an investigational ...

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Acadia Pharmaceuticals announces trofinetide new drug application for the treatment of Rett syndrome has been accepted for filing and review by US FDA

12 September 2022 - Prescription Drug User Fee Act action date set for 12 March 2023. ...

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Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alfa mannosidosis

12 September 2022 - FDA grants priority review designation with PDUFA date in the first half of 2023. ...

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InnoCare announces priority review of orelabrutinib for the treatment of relapsed or refractory marginal zone lymphoma by NMPA

6 September 2022 - InnoCare Pharma announced today that BTK inhibitor orelabrutinib has been granted priority review for the treatment of ...

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FDA grants priority review to efanesoctocog alfa for people with haemophilia A

30 August 2022 - Priority review based on pivotal data from the XTEND-1 Phase 3 study. ...

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iECURE receives FDA rare paediatric disease designation for GTP-506, an investigational gene editing product candidate for the treatment of ornithine transcarbamylase deficiency

23 August 2022 - GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC ...

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Lynparza in combination with abiraterone granted priority review in the US for patients with metastatic castration-resistant prostate cancer

16 August 2022 - First PARP inhibitor to demonstrate clinical benefit in combination with a new hormonal agent irrespective of ...

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Menarini Group’s elacestrant granted priority review by the US FDA for patients with ER+/HER2- advanced or metastatic breast cancer

11 August 2022 - US FDA has assigned a Prescription Drug User Fee Act date of 17 February 2023. ...

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