- PMLiVE

Eli Lilly and Alchemab Therapeutics enter ALS agreement worth up to $415m

The deal follows the companies’ earlier partnership to advance candidates for the neurodegenerative disease

- PMLiVE

AstraZeneca’s Calquence approved by EC for first-line mantle cell lymphoma use

The drug is now the only BTK inhibitor to be approved in the EU for first-line MCL

- PMLiVE

Alnylam’s RNAi therapeutic vutrisiran recommended by CHMP for rare heart disease

The committee has recommended the drug for both wild type and hereditary ATTR-CM

- PMLiVE

Johnson & Johnson’s Imaavy granted FDA approval for rare autoimmune disease gMG

Myasthenia gravis affects an estimated 700,000 people worldwide

- PMLiVE

FDA approves Abeona’s epidermolysis bullosa gene therapy Zevaskyn

The sheet-based treatment is expected to be available from the third quarter of this year

- PMLiVE

MHRA approves Autolus Therapeutics’ Aucatzyl to treat acute lymphoblastic leukaemia

Approximately 765 new cases of the aggressive blood cancer are diagnosed in the UK every year

- PMLiVE

Merck KGaA expands rare tumour portfolio with $3.9bn SpringWorks acquisition

SpringWorks’ Gomekli was approved by the FDA earlier this year to treat patients with NF1

- PMLiVE

Ipsen’s elafibranor shows promise in rare liver disease primary sclerosing cholangitis

The drug already holds approvals to treat primary biliary cholangitis, another rare liver disease

- PMLiVE

Pfizer’s Hympavzi granted MHRA approval to treat haemophilia A and B

More than 11,800 people were living with the genetic blood disorders in the UK in 2023/2024

- PMLiVE

FDA approves pre-filled syringe version of argenx’s immune disorder drug Vyvgart

The authorisation will give patients the flexibility to decide when and where they receive treatment

- PMLiVE

Compass Therapeutics’ tovecimig shows promise in phase 2/3 biliary tract cancer study

Around 23,000 cases of the aggressive cancer are diagnosed in the US every year

- PMLiVE

Ipsen’s Iqirvo accepted by SMC to treat rare liver disease primary biliary cholangitis

The life-long condition affects approximately 1,900 people in Scotland

- PMLiVE

Amgen’s Uplizna approved by FDA as first treatment for rare disease IgG4-RD

Immunoglobulin G4-related disease affects approximately 20,000 people in the US

- PMLiVE

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

Almost 13 out of every million people in the US are diagnosed with IgAN each year

- PMLiVE

Sanofi’s Qfitlia granted FDA approval to treat haemophilia A or B

Qfitlia is now the first therapy in the US to treat the rare bleeding disorder regardless of inhibitor status