FDA grants priority review of Nucala for patients with hypereosinophilic syndrome

27 May 2020 - An approval would give Nucala a third indication in an eosinophil driven disease. ...

Read more →

Alnylam announces U.S. FDA has granted priority review of the lumasiran new drug application for the treatment of primary hyperoxaluria type 1

26 May 2020 - PDUFA date set for 3 December 2020. ...

Read more →

Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis

21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...

Read more →

Eiger BioPharmaceuticals announces FDA acceptance of NDA for filing with priority review of Zokinvy (lonafarnib) for treatment of progeria and progeroid laminopathies

19 May 2020 - Eiger BioPharmaceuticals today announced that the U.S. FDA accepted the new drug application for accelerated review of ...

Read more →

Seelos Therapeutics receives rare paediatric disease designation for SLS-005 (trehalose) in Sanfilippo syndrome

15 May 2020 - Seelos Therapeutics today announced it has been granted rare paediatric disease designation for SLS-005 in Sanfilippo ...

Read more →

FDA grants priority review of sutimlimab, potential first approved treatment of haemolysis in adult patients with cold agglutinin disease

14 May 2020 - Sutimlimab targets C1-activated haemolysis in cold agglutinin disease. ...

Read more →

CRISPR Therapeutics and Vertex Pharmaceuticals announce FDA regenerative medicine advanced therapy designation granted to CTX001 for the treatment of severe hemoglobinopathies

11 May 2020 - CTX001 has received orphan drug designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the ...

Read more →

FDA grants paediatric disease designation for Mateon's CA4P

5 May 2020 - Treatment of stage IIB–IV melanoma due to genetic mutations that disproportionately affect paediatric patients. ...

Read more →

Immunicum receives regenerative medicine advanced therapy designation from FDA for ilixadencel in kidney cancer

6 May 2020 - Immunicum announced today that it has received regenerative medicine advanced therapy designation from the U.S. FDA for ...

Read more →

Antabio receives FDA qualified infectious disease product designation for MEM-ANT3310, a novel broad-spectrum combination therapy targeting WHO’s priority pathogens

6 May 2020 - QIDP is granted to Antabio’s MEM-ANT3310 for major hospital indications including nosocomial pneumonia. ...

Read more →

Acceleron receives PRIME Designation from EMA for sotatercept in pulmonary arterial hypertension

4 May 2020 - PRIME designation comes just three weeks after US FDA granted sotatercept breakthrough therapy designation. ...

Read more →

U.S. FDA accepts for priority review Bristol Myers Squibb’s application for CC-486 for maintenance treatment of adult patients in remission with acute myeloid leukaemia

1 May 2020 - Application based on results from the Phase 3 QUAZAR-AML-001 study. ...

Read more →

Novartis Kymriah receives FDA regenerative medicine advanced therapy designation in follicular lymphoma

22 April 2020 - US regulatory filing for Kymriah in relapsed/refractory follicular lymphoma anticipated in 2021. ...

Read more →

FDA accepts for priority review biologics license application for REGN-EB3 to treat Ebola

16 April 2020 - REGN-EB3 developed via same rapid response platform currently being leveraged to develop novel COVID-19 antibody therapy. ...

Read more →

Merck receives priority review from FDA for second application for Keytruda (pembrolizumab) based on biomarker, regardless of tumour type

7 April 2020 - Supplemental biologics license application accepted for Keytruda monotherapy in patients whose tumours are tumour mutational burden-high who ...

Read more →