- PMLiVE

Sobi’s Aspaveli receives expanded EC approval for rare blood disorder PNH

The C3 therapy can now be used as a first-line treatment for paroxysmal nocturnal haemoglobinuria

- PMLiVE

AstraZeneca’s Voydeya granted EC approval to treat rare blood disease PNH

The factor D inhibitor has been authorised for use alongside the company’s complement C5 inhibitors

- PMLiVE

Rare diseases – raising awareness, listening to patients, making a difference

PME speaks to Lorna Pender, Global Patient Engagement Lead at Clinigen, about the treatment options available to patients with rare diseases and the barriers that are hindering access

- PMLiVE

Ipsen and Skyhawk announce $1.8bn partnership to advance rare neurological disease therapies

The agreement gives Ipsen an option to receive exclusive global rights to two candidates

- PMLiVE

Novartis shares positive late-stage results for Fabhalta in rare kidney disease IgA nephropathy

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

- PMLiVE

MHRA approves TMC Pharma’s Nulibry to treat rare genetic disease MoCD type A

Molybdenum cofactor deficiency is estimated to affect one in 100,000 to 200,000 newborns worldwide

- PMLiVE

Single genetic test could replace current standard for diagnosing rare developmental disorders

The single genetic test could potentially replace the current standard and reduce costs for the NHS

- PMLiVE

Sobi shares positive phase 3 results for Doptelet in paediatric patients with rare blood disorder ITP

Immune thrombocytopenia is estimated to affect up to 100 people per million

- PMLiVE

AstraZeneca’s Ultomiris receives FDA approval to treat rare autoimmune disease NMOSD

Neuromyelitis optica spectrum disorder is estimated to affect approximately 6,000 adults in the US

- PMLiVE

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

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

- PMLiVE

Takeda’s HyQvia receives MHRA approval for rare neurological disorder CIDP

The therapy offers patients with CIDP a typical dosing interval of four weeks

- PMLiVE

Takeda’s Iclusig combination granted FDA accelerated approval for rare form of leukaemia

The drug is now the first targeted therapy approved as a frontline treatment for Ph-positive ALL

- PMLiVE

FDA approves Mirum’s Livmarli to treat cholestatic pruritus in rare liver disease patients

Progressive familial intrahepatic cholestasis affects one in every 50,000 to 100,000 births in the US and Europe

- PMLiVE

AstraZeneca expands rare disease pipeline with $1.05bn Amolyt Pharma acquisition

The deal gives the company access to a late-stage candidate being evaluated for hypoparathyroidism

- PMLiVE

Almirall gains rights to Eloxx’s rare dermatological disease asset in deal worth over $470m

ZKN-013 is designed to overcome nonsense mutations in rare skin diseases such as junctional epidermolysis bullosa