- PMLiVE

MHRA approves Pharming’s Joenja as first drug for rare immune disease APDS

Up to 40 people in the UK are affected by activated phosphoinositide 3-kinase delta syndrome

- PMLiVE

FDA grants rare paediatric disease designation to Enterprise’s cystic fibrosis candidate

The genetic disease is estimated to affect more than 100,000 people globally

- PMLiVE

Laying the groundwork for rare disease therapies

Addressing the challenges of convincing payers that the therapy meets an unmet need

- PMLiVE

AstraZeneca’s Fasenra receives FDA approval to treat rare inflammatory disease EGPA

Eosinophilic granulomatosis with polyangiitis affects approximately 15,000 people in the US

- PMLiVE

Sanofi, RadioMedix and Orano Med partner to develop rare cancer therapy in deal worth €320m

AlphaMedix is currently being evaluated as a treatment for neuroendocrine tumours

- PMLiVE

FDA grants full approval to Travere’s Filspari for rare kidney disease IgA nephropathy

The regulator’s decision expands the drug’s indication to cover all patients who are at risk of disease progression

- PMLiVE

MSD’s Welireg recommended by NICE to treat rare von Hippel-Lindau disease

Approximately one in every 70,000 people are affected by the tumour-causing genetic disease

- PMLiVE

Novartis’ Fabhalta recommended by NICE to treat rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately ten to 20 people per million worldwide

- PMLiVE

Breast cancer drug found to help slow growth of rare childhood brain tumour

Researchers discovered that targeting the CDK6 protein could make tumour cells vulnerable to treatment

- PMLiVE

Roche’s monoclonal antibody PiaSky approved by EC to treat rare blood disorder PNH

The drug is now the first monthly subcutaneous treatment authorised for use in this patient population

- PMLiVE

Sobi and Apellis’ pegcetacoplan shows promise in phase 3 rare kidney disease study

There are currently no treatments that target the underlying cause of C3G or IC-MPGN

- PMLiVE

Novartis’ Fabhalta granted FDA approval for rare kidney disease IgA nephropathy

Approximately 25 people per million worldwide are diagnosed with IgAN every year

- PMLiVE

Calliditas announces promising results for NOX enzyme inhibitor in rare liver disease

Primary biliary cholangitis is estimated to affect approximately 140,000 patients in the US

- PMLiVE

FDA approves Samsung Bioepis’ Soliris biosimilar Epysqli for rare blood disorders

Epysqli has been authorised to treat paroxysmal nocturnal haemoglobinuria and atypical haemolytic uraemic syndrome

- PMLiVE

FDA establishes rare disease innovation hub to accelerate treatment development

More than 30 million people in the US are currently affected by a rare disease