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
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
FDA approves Apellis’ Empaveli for two rare kidney diseases
C3G and primary IC-MPGN are estimated to affect 5,000 people in the US
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
Novo Nordisk’s Alhemo approved by FDA for expanded haemophilia use
The rare bleeding disorder affects approximately 800,000 people globally
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
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
EC approves SpringWorks Therapeutics’ Ezmekly to treat rare genetic disorder NF1
Neurofibromatosis type 1 affects an estimated 135,000 people in the EU
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
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
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
Vertex’s triple combination cystic fibrosis therapy Alyftrek recommended by NICE
The rare inherited disease affects approximately 11,000 people in the UK
Vertex Pharmaceuticals’ cystic fibrosis therapy Alyftrek granted EC approval
The rare inherited disease affects more than 109,000 people worldwide
EC approves Jazz Pharmaceuticals’ Ziihera for advanced biliary tract cancer
Approximately 26% of biliary tract cancer patients globally have HER2-positive disease
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