Keeping tabs on pharma: World’s first GMO heart transplant, new insomnia drug

World’s first genetically-modified pig’s heart transplant is a success 

A US man has become the world's first person to receive a heart transplant from a genetically-modified pig thanks to advances in gene editing tools. David Bennett, 57, underwent the seven-hour operation at the University of Maryland Medical Center in Baltimore, US. 

Bennett had not been eligible for a human heart because he had advanced health problems. Instead he consented to the transplant of a heart from a genetically modified pig created by Revivicor, a subsidiary of United Therapeutics. 

Revivicor made 10 alterations to the pig's genes, including inserting six human genes into its genome to prevent rejection of the organ once transplanted. So far the operation has been deemed a success. 

The transplant follows announcements from Bayer and Pfizer in December 2021 on their new partnerships with Mammoth Biosciences and Beam Therapeutics respectively, two gene editing companies. 

New drug to treat insomnia approved by FDA 

Swiss biotech firm Idorsia has received approval from the US Food and Drug Administration (FDA) for its insomnia drug Quviviq. 

The FDA’s approval signifies an important milestone for Idorsia and its husband and wife leadership team, who have been working on the insomnia pill for more than two decades. Martine Clozel, MD and Chief Scientific Officer of Idorsia, explained: “After more than 20 years of research and a progressive understanding of the role of orexin in sleep-wake balance and of the potential of orexin receptor antagonism, we designed daridorexant to help address several issues people with insomnia face. 

“Daridorexant properties include a potent inhibition of both orexin receptors, a rapid absorption for sleep onset, and a pharmacokinetic profile such that around 80 percent of daridorexant has been eliminated after a night of sleep to help minimize residual effects.”

The FDA has recommended that Quviviq be classified as a controlled substance and it is expected to be available to patients from May 2022. In the meantime a high-profile publicity campaign featuring Hollywood actress Jennifer Aniston is already underway.

Treatment for children with cystic fibrosis approved in Europe

The European Commission has granted Vertex approval for the label extension of Kaftrio to treat cystic fibrosis (CF) in patients aged six to 11 years. 

The combination of ivacaftor, tezacaftor and elexacaftor has shown good results in people aged 12 and above with CF, and it is hoped the treatment will help slow down the advancement of the disease in younger patients. “CF is a progressive disease, in which symptoms and organ damage manifest very early in life,” said Professor Marcus A. Mall, Head of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. “As a physician, I welcome the approval of this medicine for this younger age group, as it will help us treat eligible children with CF as early as six years old.”

To be eligible patients must also have a f508del mutation. Patients in Austria, Northern Ireland, Denmark and Germany will have access to the expanded indication for Kaftrio following the European Commission’s regulatory approval, and it has also been approved for use in New Zealand and Switzerland, where it is known as Trikafta. 

Also in the news

• Early trial results for a new spinal-cord injury treatment are showing "a real reason for hope," NervGen Pharma’s CEO has said. 
• Biogen's controversial Alzheimer's drug could be restricted to clinical trials if a new proposal from the US Centers for Medicaid and Medicare Services to limit its use comes into effect. In a statement on its website, Biogen announced it intends to respond to the draft decision within 30 days. 
• A new hub dedicated to the study and treatment of Alport syndrome – a disease that damages the tiny blood vessels in kidneys – is set to launch in England. 
• Sanofi and Merck have signed major deals with two startups to harness deep learning and AI in drug discovery. In the case of Sanofi’s $US 100m agreement with Exscientia, up to 15 new drugs could be in the pipeline.