Nearly four months after Intellia Therapeutics – along with partner Regeneron Pharmaceuticals – reinvigorated the CRISPR space with the first-ever clinical evidence that CRISPR gene editing inside the body can be safe and effective, the asset in question was granted Orphan Drug Designation by the U.S. Food and Drug Administration.
CureVac NV said on Oct. 12 the company will give up on the biotechnology firm’s first-generation Covid-19 vaccine candidate and instead focus on collaborating with GSK to develop improved mRNA vaccine technology.
The U.S. Food and Drug Administration Antimicrobial Drugs Advisory Committee (AMDAC) voted unanimously to recommend use of Takeda Pharmaceutical Company Limited’s maribavir (TAK-620) for the treatment of refractory cytomegalovirus (CMV) infection and disease withgenotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients.
U.S.-based Biomarin Pharmaceutical Inc.’s treatment for one of the most common forms of dwarfism received clearance from the European Commission, becoming the first therapy to get an approval in the region for achondroplasia.
AstraZeneca said on Aug. 26 a late-stage trial showed the company’s medicine for a rare disease that causes accumulation of copper in the body was three times more successful in helping remove deposits from tissues compared with standard care.
The U.S. drug regulator declined to approve Sesen Bio Inc.’s lead therapy candidate for a type of bladder cancer, the company said on Aug. 13, sending its shares down more than 80 percent.
Three new conditions reported by a small number of people after vaccination with Covid-19 shots from Pfizer and Moderna are being studied to assess if they may be possible side effects, Europe’s drugs regulator said on Aug. 11.
Albireo Pharma, a spinout of AstraZeneca, won regulatory approval for the first drug aimed at pruritus in patients with all subtypes of progressive familial intrahepatic cholestasis (PFIC).
The head of the EU drug regulator’s Covid-19 task force said on June 13 that AstraZeneca’s coronavirus vaccine had a favorable risk-benefit profile for all age groups and particularly for those aged over 60.
Europe’s drug regulator on June 11 identified another rare blood condition as a potential side effect of AstraZeneca’s Covid-19 vaccine and said it was looking into cases of heart inflammation after inoculation with all coronavirus shots.