A CGT burst
In less than two months time, the U.S. has seen four new cell and gene therapy approvals — putting the agency on track for a record-setting year.
The run started in mid-May when the FDA approved Krystal Biotech's topical gene therapy for the repeated treatment of a serious, rare skin disease. The drug, branded Vyjuvek, is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa.
Then in June, within a week's span, the CGT sector notched three more approvals.
On June 22, Sarepta Therapeutics announced that the FDA had granted accelerated approval for SRP-9001, to be sold as Elevidys. The one-time treatment is a gene therapy based on adeno-associated virus, approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy.
Days later, on June 28, the agency approved CellTrans’ Lantidra, marking the first allogeneic pancreatic islet cell therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes.
The next day, the FDA approved BioMarin Pharmaceutical's Roctavian, the first and only gene therapy for adults with severe hemophilia A.
Last year, the U.S. saw a record-setting five CGT commercial approvals, including the first gene therapy authorized for hemophilia B. During Phacilitate's Advanced Therapies Week back in January, 2022 was commemorated as 'the year the commercial reality of CGT became apparent' — but 2023 might surpass it.
With the addition of Gamida Cell’s allogeneic cell therapy, Omisirge, approved in April, 2023 now has matched 2022 with five approvals classified as CGT by the agency.
It's a good showing for CGT companies — and CBER, which has been plagued with staffing and retention issues, consistently losing staff to the higher paying private sector. But those who will benefit most from increased approvals are patients, so here's to a record-shattering year!
—Karen Langhauser
Insider trading drama
U.S. Attorney Damian Williams has had a busy few weeks. In a statement released last Thursday, Williams outlined insider trading charges in four separate cases, two of which involved pharma employees.
This week, we wrote about former Pfizer employee Amit Dagar being implicated in insider trading related to Pfizer's Paxlovid clinical trials. Dagar, who had access to confidential trial data, purchased call options in Pfizer stock before the positive trial results were publicly announced. He tipped off another person who also bought similar options.
Both individuals, along with another person identified as "Individual-1," sold their options after Pfizer's announcement, resulting in profits of approximately $350,000. Dagar has now been arrested and charged with securities fraud and conspiracy.
In another case, senior Alexion employee Joseph M. Dupont allegedly shared confidential information about Alexion’s upcoming acquisition of Portola Pharmaceuticals with a friend. That friend told another friend who told another friend and insider details were shared with relatives and colleagues.
According to the SEC's complaint, the defendants collectively gained over $2.3 million through their insider trading activities involving stocks and call options. Other traders who were tipped by friends saw profits of an additional $1.7 million.
Following their successful trades, two of the men implicated texted each other in Russian, saying, "Let's hope our golden goose will continue laying golden eggs!"
The SEC is seeking legal remedies, including permanent injunctions against the defendants to prevent further violations of securities laws. Aiming to recover the defendants' gains through disgorgement and prejudgment interest, the SEC is also requesting a permanent ban on the defendants serving as officers or directors of registered companies due to their involvement in illegal insider trading.
—Andrea Corona
