- PMLiVE

DEBRA and Queen Mary partner to advance epidermolysis bullosa drug development

Around 500,000 people worldwide are affected by the group of rare genetic skin conditions

- PMLiVE

FDA approves Jazz Pharmaceuticals’ Modeyso as first drug for ultra-rare brain cancer

H3 K27M-mutant diffuse midline glioma affects an estimated 2,000 people in the US every year

- PMLiVE

FDA approves Apellis’ Empaveli for two rare kidney diseases

C3G and primary IC-MPGN are estimated to affect 5,000 people in the US

- PMLiVE

FDA approves PTC Therapeutics’ Sephience to treat rare metabolic disorder PKU

Phenylketonuria affects one in every 13,500 to 19,000 newborns in the US

- PMLiVE

Novo Nordisk’s Alhemo approved by FDA for expanded haemophilia use

The rare bleeding disorder affects approximately 800,000 people globally

- PMLiVE

Roche’s Evrysdi tablets approved by MHRA to treat spinal muscular atrophy

It is hoped the new formulation will offer patients greater freedom and flexibility compared to the liquid version

- PMLiVE

Biogen’s Qalsody granted MHRA approval to treat rare form of motor neurone disease

Mutations in the SOD1 gene are responsible for approximately 2% of all cases of the disease

- PMLiVE

EC approves SpringWorks Therapeutics’ Ezmekly to treat rare genetic disorder NF1

Neurofibromatosis type 1 affects an estimated 135,000 people in the EU

- PMLiVE

NICE recommends BeOne’s Brukinsa to treat mantle cell lymphoma on NHS

Approximately 600 people are affected by the form of non-Hodgkin lymphoma every year in the UK

- PMLiVE

Alnylam’s Amvuttra approved by MHRA to treat rare heart disease ATTR-CM

The drug has been authorised to treat both wild-type and hereditary forms of the disease

- PMLiVE

MHRA approves KalVista Pharmaceuticals’ Ekterly to treat hereditary angioedema attacks

Ekterly is now the first and only oral on-demand treatment for HAE to be approved by the UK regulator

- PMLiVE

Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE

The rare inherited disease affects approximately 11,000 people in the UK

- PMLiVE

Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval

The rare inherited disease affects more than 109,000 people worldwide

- PMLiVE

EC approves Jazz Pharmaceuticals’ Ziihera for advanced biliary tract cancer

Approximately 26% of biliary tract cancer patients globally have HER2-positive disease

- PMLiVE

UCB shares promising results for fenfluramine in ultra-rare form of epilepsy

CDKL5 deficiency disorder occurs in approximately one in 40,000 to 60,000 live births