- PMLiVE

Pfizer shares positive phase 3 results for haemophilia A and B therapy Hympavzi

Around 20% and 3% of haemophilia A and B patients, respectively, develop inhibitors

- PMLiVE

Boehringer’s Spevigo recommended by NICE for generalised pustular psoriasis

The rare form of psoriasis can lead to serious complications such as sepsis and heart failure

- PMLiVE

FDA approves CSL’s Andembry to prevent hereditary angioedema attacks

The genetic disorder affects approximately one in 10,000 to one in 50,000 people worldwide

- PMLiVE

AstraZeneca’s Ultomiris shows promise in rare post-transplant complication

There are currently no approved treatment options for HSCT-TMA

- PMLiVE

Novartis shares positive late-stage results for Fabhalta in rare blood disorder PNH

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

- PMLiVE

FDA approves Nuvation Bio’s TKI inhibitor Ibtrozi to treat rare form of lung cancer

ROS1-positive non-small cell lung cancer accounts for approximately 2% of new NSCLC cases

- PMLiVE

Alnylam’s Amvuttra granted EC approval to treat rare heart disease ATTR-CM

The RNAi therapeutic has been authorised to treat both wild type and hereditary forms of the disease

- PMLiVE

Sobi/Apellis’ pegcetacoplan shows sustained efficacy in phase 3 kidney disease study

C3G and primary IC-MPGN are estimated to affect 5,000 people in the US and up to 8,000 in Europe

- PMLiVE

Otsuka shares promising phase 3 results for sibeprenlimab in rare kidney disease IgAN

The trial will continue to evaluate change in kidney function over two years

- PMLiVE

Dermaliq and DEBRA partner to advance epidermolysis bullosa drug delivery

Approximately 500,000 people worldwide are affected by the rare genetic skin disorders

- PMLiVE

Merck’s Welireg granted FDA approval to treat rare tumour types PPGL

Up to 2,000 new cases of pheochromocytoma or paraganglioma are diagnosed in the US every year

- PMLiVE

Alnylam shares phase 3 results for Amvuttra in rare heart disease ATTR-CM

Transthyretin amyloid cardiomyopathy is estimated to affect over 300,000 people worldwide

- PMLiVE

Takeda’s Adzynma approved by MHRA for rare blood clotting disorder cTTP

The enzyme replacement therapy is now the first treatment approved in the UK for this patient population

- PMLiVE

FDA approves Verastem Oncology’s combination therapy for rare ovarian cancer type

Approximately 6,000 to 8,000 women in the US are living with low-grade serous ovarian cancer

- PMLiVE

GSK shares promising phase 3 results for linerixibat in PBC-related itch

Pruritus is experienced by up to 90% of patients with the rare liver disease