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FDA awards over $38m for advancement of rare disease treatments

Grants will support clinical trials, natural history studies and regulatory science tools

Biomarin

BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

- PMLiVE

Pfizer/BioNTech BA.4/BA.5 COVID-19 booster approved for emergency use by FDA in children five to 11 years

The doses will be shipped immediately, pending recommendation from the CDC

regeneron headquarters

Regeneron’s application for Eylea in retinopathy of prematurity accepted for FDA priority review

The rare eye disease often impacts infants who are born before 31 weeks of pregnancy

- PMLiVE

Genentech shares positive two-year data for Evrysdi in spinal muscular atrophy study

The study enrolled the ‘broadest and most diverse’ patient population ever studied in an SMA trial

- PMLiVE

Eli Lilly granted fast track designation by FDA for obesity treatment

The company plans to initiate a rolling submission of a new drug application for tirzepatide this year

- PMLiVE

InflaRx seeks FDA authorisation for vilobelimab for critically ill COVID-19 patients

A phase 3 study showed that the antibody reduced the all-cause mortality rate after 28 days

- PMLiVE

Amylyx’s Relyvrio receives FDA approval for ALS treatment

Relyvrio was shown to significantly slow physical decline in people living with ALS

- PMLiVE

Sanofi and Regeneron’s Dupixent approved by FDA for prurigo nodularis

The two phase 3 trials showed a meaningful reduction in itching

- PMLiVE

Pfizer and BioNTech seek FDA authorisation for COVID-19 booster for children

An application will also be submitted to the European Medicines Agency to extend the marketing authorisation for this age group

- PMLiVE

Lilly’s Retevmo approved by FDA for RET fusion+ advanced/metastatic solid tumours

Results demonstrated an overall response rate of 44% across multiple tumour types

- PMLiVE

bluebird bio’s Skysona approved by FDA to treat cerebral adrenoleukodystrophy

The gene therapy was shown to slow the progression of neurologic dysfunction

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