Bluebird’s new gene therapy among three new EMA OKs ahead of FDA
Ahead of a usually speedy FDA, the European Medicines Agency on Friday recommended marketing authorizations for bluebird bio’s new gene therapy for children with a rare, inherited neurological disease, GlaxoSmithKline and Vir’s monoclonal antibody treatment for Covid-19, and a new treatment for a rare liver disease.
In the case of bluebird’s Skysona (elivaldogene autotemcel), the gene therapy is for those younger than 18 who have what’s known as early Cerebral Adrenoleukodystrophy (CALD). If untreated, nearly half of those with CALD die within 5 years of symptom onset, EMA said.
EMA’s recommendation is based on a single-arm trial in 32 boys with CALD (the disorder is almost exclusively seen in males) who received the one-time treatment, compared to 59 boys who had a stem cell transplantation.
“An analysis conducted after 24 months from the infusion on 30 subjects enrolled in the study concluded that for 27 of them (90%) treatment with Skysona preserved motor function and communication ability and improved survival when compared to untreated patients at an early stage of cerebral disease,” the EMA said.
Bluebird bio said it is currently on track to submit the Biologics License Application to the FDA by mid-2021.
Richard Colvin, interim CMO at bluebird, said in a statement, “This positive opinion from the CHMP marks the first regulatory approval recommendation for any gene therapy for CALD, bringing us closer to a one-time, durable treatment option that stabilizes neurological disease while reducing the risk of the serious immune complications associated with allogeneic stem cell transplantation (allo-HSCT), which is the only therapeutic option for children with this devastating disease.”
GSK and Vir mAb
The EMA on Friday also concluded that GSK’s and Vir’s mAb Covid-19 treatment, known as sotrovimab, can be used to treat confirmed Covid-19 in adults and adolescents who don’t require supplemental oxygen therapy and are at risk of progressing to severe Covid-19.
EMA said an interim analysis of the infusion indicated that sotrovimab reduced the risk of hospitalization for more than 24 hours or death by 85% compared with placebo. Hospitalization for more than 24 hours or death occurred in 1% (3 out of 291) of patients who received sotrovimab and 7% (21 out of 292) of those who received placebo.
EU member states can use the positive scientific opinion when making national decisions about the early use of sotrovimab prior to EMA marketing authorization. A rolling review of the treatment is ongoing, as of May 7, and will be the basis for a marketing authorization, EMA said.
The companies said they also submitted an emergency use authorization application for the mAb to the FDA, and Health Canada is also reviewing it.
Albireo’s rare liver disease drug
The EMA also recommended granting marketing authorization to Albireo’s Bylvay (odevixibat) for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients aged 6 months or older.
PFIC is a rare, life-threatening liver disease and only about half of the children affected by the disease survive beyond the age of 10 years.
Results from a Phase III trial showed a significant reduction in serum bile acids accompanied by a significant reduction in pruritus in those treated with odevixibat.
“These results were maintained in an ongoing, long-term open-label follow-up study. Hepatic parameters and fibrosis scores were improving or were stable for the duration of the study (max. 72 weeks). However, more data are needed to determine if odevixibat can delay disease progression and the need for liver transplantation. The CHMP therefore requested a registry-based efficacy study as a follow-up,” EMA said.
The FDA has until July 20 to decide on whether to approve the drug, and the company said an advisory committee meeting is not expected. If approved, Albireo also would win a lucrative rare pediatric disease priority review voucher.