HOT & NOT

AstraZeneca’s Forxiga (dapagliflozin) has been approved by the European Commission for the treatment of chronic kidney disease (CKD) in the European Union. The Medicines and Healthcare products Regulatory Agency (MHRA) has similarly approved a licence extension for Forxiga in Great Britain. Both decisions are based on positive results from the DAPA-CKD Phase III trial, which demonstrated that dapagliflozin plus standard care showed a significant reduction in all-cause mortality in a renal outcomes trial in patients with CKD.

Albireo Pharma’s Bylvay (odevixibat) has received approval on both sides of the Atlantic, scoring authorisations in the EU and US for the treatment of all subtypes of progressive familial intrahepatic cholestasis (PFIC). Bylvay is the first drug to be approved for PFIC – prior to its authorisation, only surgical options including biliary diversion surgery (BDS) and liver transplantation have been available to these patients. The approvals are supported by data from the Phase III PEDFIC 1 and PEDFIC 2 trials.

The UK’s National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for Incyte Biosciences UK’s Pemazyre (pemigatinib) for the treatment of the rare bile duct cancer cholangiocarcinoma (CCA). NICE’s guidance recommends Pemazyre for the treatment of CCA with fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy. CCA is a rare type of liver cancer that forms in the bile duct and is classified based on its origin.

The Scottish Medicines Consortium (SMC) was unable to accept Pfizer’s Vyndaqel (tafamidis) for the treatment of the rare heart condition transthyretin amyloidosis. The SMC said it was unable to accept Vyndaqel as the evidence provided by Pfizer was ‘not strong enough’ to satisfy the committee that the treatment offers value for money to NHS Scotland. NICE also rejected Vyndaqel after determining that cost-effectiveness estimates for the drug were higher than what would be considered an acceptable use of NHS resources.

NICE has not recommended Orchard Therapeutics’ gene therapy Libmeldy (autologous CD34+ cells encoding the ARSA gene) for the treatment of metachromatic leukodystrophy (MLD) in children. In a statement, NICE said that although clinical trial evidence suggests Libmeldy improves motor and cognitive function and could correct the enzyme deficiency caused by MLD, only short-term evidence is available. NICE said that, as such, assumptions about the long-term stabilisation of symptoms are ‘very uncertain’ with Libmeldy treatment.

NICE has decided not to recommend Janssen’s Zytiga (abiraterone) after reviewing its previous decision to reject the drug. In June 2020, NICE initially rejected Zytiga as a first-line treatment for newly diagnosed, advanced prostate cancer. Following this initial rejection, NICE announced that it would reconsider its decision. However, the cost-effectiveness watchdog has now concluded that it will not recommend Zytiga. In a statement, The Institute of Cancer Research, London (ICR) said it is ‘disappointed’ by NICE’s final decision.