aromatic L-amino acid decarboxylase deficiency
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
J&J’s Darzalex recommended by CHMP to treat smouldering multiple myeloma
If approved, Darzalex would be the first therapy approved in the EU for SMM
Organon/Henlius announce EMA validation for Perjeta biosimilar HLX11
The reference medicine holds approvals for multiple HER2-positive breast cancer indications
Rare diseases: Novartis’ Fabhalta recommended by CHMP for C3 glomerulopathy
Approximately one to two people per million worldwide are diagnosed with the ultra-rare kidney disease every year
Rare diseases: CHMP recommends Krystal’s Vyjuvek for dystrophic epidermolysis bullosa
There is currently only one treatment authorised for the ultra-rare genetic blistering disease
CHMP recommends Bavarian Nordic’s chikungunya vaccine for individuals aged from 12 years
Approximately 480,000 cases of the mosquito-borne viral disease were reported globally in 2024
Gilead’s seladelpar recommended by CHMP to treat primary biliary cholangitis
The rare liver disease affects approximately 15 per 100,000 people in Europe
GSK’s Jemperli receives positive CHMP opinion for expanded endometrial cancer use
Approximately 121,000 people are diagnosed with primary advanced or recurrent endometrial cancer each year in Europe
Novartis gains rights to PTC’s Huntington’s programme in deal worth up to $2.9bn
The genetic disorder affects one in every 10,000 to 20,000 people in the US
MHRA urges companies to prepare for Windsor Framework for medicines
The new UK-wide arrangements for medicines will come into effect on 1 January 2025
Biogen/Eisai’s Alzheimer’s drug lecanemab receives CHMP recommendation
The neurodegenerative disorder affects an estimated 6.9 million people in Europe
Novartis’ Kisqali receives CHMP recommendation for early breast cancer patients
Approximately 70% of all breast cancer patients have HR-positive/HER2-negative disease