Alexion wins priority review for Ultomiris' aHUS indication; FDA expands approval of Vertex's Symdeko
→ Alexion $ALXN has scored a speedy review for Ultomiris for patients with atypical hemolytic uremic syndrome (aHUS) after posting positive data from a pivotal study in January. The drug is the rare disease company’s shot at protecting its blockbuster blood disorder franchise that is currently centered around its flagship drug, Soliris, which is a complement inhibitor typically administered every two weeks. Ultomiris has a similar mechanism of action but requires less-frequent dosing — every eight weeks. The decision date has been set to October 19. Late last year, Ultomiris secured approval for nocturnal hemoglobinuria (PNH) patients.
→ The label on Vertex‘s $VRTX CF treatment Symdeko (tezacaftor/ivacaftor and ivacaftor) was expanded for use in children with cystic fibrosis aged between 6 and 11 years with certain mutations on Friday. “We’ve seen the clinical impact of SYMDEKO in people with CF aged 12 years and above, and this approval marks a crucial milestone for patients ages 6 through 11 years who may benefit from CFTR modulation, enabling us to treat the basic defect in CF at an earlier stage of disease,” said Seth Walker, of University Hospitals of Cleveland, Cleveland Medical Center, Rainbow Babies and Children’s Hospital.
→ Kymab has agreed to put its platform tech to use for the UK medical charity LifeArc, gaining a new investor who’s taking a $30 million equity stake in the biotech. LifeArc will get its hands on Kymab’s antibody discovery technology, and the biotech also stands to earn royalties on any new drugs that come out of the deal. “I am delighted that LifeArc, with its focus on transformational medical research which has led to drugs such as Keytruda, has chosen our IntelliSelect platforms for their future antibody discovery programmes,” noted Kymab CEO Simon Sturge.