Forecast 2022: Cell and gene therapy manufacturers push back regulatory and payers hurdles to create high hopes for 2022



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For developers of cell and gene therapy, the path to market often seems strewn with potholes. The FDA has rejected BioMarin Pharmaceutical’s gene therapy to treat hemophilia A in 2020, citing the need for more sustainability data and dropping the company’s shares by more than 20% in one day. In November of this year, the FDA delayed a long-awaited decision on the multiple myeloma candidate CAR-T cilta-cel from Legend Biotech and Johnson & Johnson, causing Legend’s shares to drop nearly 8%.

Now, these companies are preparing for the FDA to finally make its decisions – and they are making those preparations with optimism. Biomarin plans to submit new data on its gene therapy, Roctavian, to the FDA in 2022, setting up potential approval by the end of the year. And, at this year’s American Society of Hematology (ASH) meeting, Legend and J&J rolled out such positive survival data from their cilta-cel trial that some analysts have suggested – if approved in February. as expected – this could have an advantage over Bristol Myers Squibb’s rival, CAR-T Abecma.

A tough journey to commercialization is normal for the cell and gene therapy course, but for companies that endure the bumps, the payoff can be significant. That’s why, despite the setback, not only regulators but payers as well, several companies are expected to generate high revenues from their cell and gene therapies in 2022. At the same time, they will be working behind the scenes. to increase profitability by improving the complex manufacturing processes required to support these expensive therapies.

“There is a high level of investment in people, monitoring quality and safety, and the effort to deepen the science,” said Ken Mills, CEO of Regenxbio, maker of viral vectors for therapy. genius, in an interview. Upcoming launches and continued global deployments of previously approved products will bring cell and gene therapy “more into public consciousness in 2022, and it will only continue to grow,” he predicted.

There are five FDA-approved CAR-T treatments for blood cancers and two gene therapies to treat rare diseases currently on the market in the United States. .

RELATED: ASH: Bristol Myers’ Breyanzi and Gilead’s Yescarta Join Race to Move CAR-T Therapy to Earlier Lymphoma

One of the biggest races to watch in the cell therapy arena will be the one between Gilead Sciences’ Yescarta and Bristol Myers Squibb’s Breyanzi, both of whom are preparing to move their CAR-T’s to earlier treatment lines in the large B cell lymphoma (LBCL). At ASH, both companies have released impressive data from their second-line trials, but Gilead could have the upper hand given its three-year lead in the market, analysts said. Gilead expects to hear from the FDA on a second line label extension in April.

“We believe that the totality of the data will help increase the uptake of CAR-T therapy for [Gilead], and expansion under the second line… could double the market opportunity, ”RBC analyst Brian Abrahams said in a note to investors at the ASH meeting. RBC estimates that Gilead’s total cell therapy revenue – it also markets the CAR-T Tecartus – will grow from $ 871 million in 2021 to $ 1.1 billion in 2022.

In the gene therapy market, Luxturna from Roche for hereditary retinal dystrophy and Zolgensma from Novartis for spinal muscular atrophy (SMA) are advancing with Zolgensma showing steady growth so far. Jefferies analysts estimate Zolgensma’s sales will grow from $ 1.3 billion in 2021 to $ 1.6 billion in 2022 and peak at $ 2.75 billion.

But reimbursement remains a challenge for all manufacturers of gene therapy. Spark, which was acquired by Roche in 2019, made waves with its initial list price of $ 425,000 per eye for Luxturna. Novartis left the door with Zolgensma at a cost of $ 2.1 million. The two companies have embarked on alternative payment models – Novartis has set the bar with a results-based agreement for its CAR-T, Kymriah, with the Centers for Medicare & Medicaid Services – but the impact of such agreements on use remains a question mark.

Novartis CEO Vasant Narasimhan told the company’s third quarter earnings conference call that Zolgensma is seeing strong adoption in the United States, Germany, Italy and the United Kingdom, and he predicted that repayment agreements in Russia and in smaller European countries would fuel overseas sales. “So if we look to the future, we see four key drivers of future growth,” he said, citing new markets, increasing rates of newborn screening for SMA, a study in Europe which “aims to build confidence in the value of Zolgensma across the EU label” and the product’s intrathecal dosing potential.

But not all manufacturers of gene and cell therapy are so optimistic. In August, bluebird bio announced that it would end its European operations after failing to reach a price agreement with authorities in several countries for Zynteglo, a gene therapy which received conditional approval from the European Agency. drugs to treat beta thalassemia in 2019. CEO Nick Leschly said on a conference call with investors at the time that the European system was “broken.”

RELATED: Cost Watching Body ICER To Bless Some High-Priced Gene And Cell Therapies, But Only With Strong Evidence Of Benefits: Analyst

Bluebird will be one of the gene therapy developers to watch in 2022, as Zynteglo for beta thalassemia is under FDA review. The company will be in a tight race with CRISPR Therapeutics and Vertex, which plan to file their genetically engineered therapy, CTX001, for approval by the end of next year.

Other companies that could see their gene therapies explode in 2022? UniQure has bounced back from a safety scare with its hemophilia B treatment, AMT-061. She and her global marketing partner, CSL Behring, could seek approval in the United States and Europe in 2022. And three potential gene therapies for Duchenne muscular dystrophy, from Pfizer, Sarepta and Solid Bio, could yield crucial data l ‘next year.

In addition, three companies are in a tight race to bring gene therapies to the market for Fabry disease: 4D Molecular Therapeutics, Sangamo and Freeline Therapeutics. They all published positive clinical data in the fall. As Evercore analysts said in a note to clients at the time, it is “very encouraging to see these programs evolve, with a lot to learn as they evolve and a lot more potential applications. as the profile of each vector / platform is developed “.

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