Upcoming FDA decisions to watch

Aug. 22, 2022
New drugs with blockbuster potential might not be far from market

The second half of 2022 is gearing up to be a busy time for the U.S. FDA. With PDUFA dates and highly-anticipated regulatory filings looming in the near future, new drugs with blockbuster potential might not be far from market.

1. Bristol Myers Squibb’s next-gen autoimmune drug

Psoriasis is a chronic, immune disorder that affects at least 100 million people around the world each year. Offering a new option for patients living with the illness, Bristol Myers Squibb’s deucravacitinib works by selectively targeting TK2 and inhibiting the signaling of cytokines that play a role in pathogenesis of a range of immune diseases.

The potential first-in-class TYK2 inhibitor is currently in trials against indications in psoriasis, psoriatic arthritis, inflammatory bowel disease and a common type of lupus. Recently, BMS reported positive data from its POETYK PSO trial — which evaluated the drug in comparison to a placebo and Amgen’s Otezla —  sharing that after a year, 58.7% of patients reported having clear or almost clear skin.

When the company announced the FDA’s acceptance of their New Drug Application last year, BMS’ senior vice president of Immunology, Jonathan Sadeh, touted the drug as a potential game-changer. “Findings from the pivotal POETYK-PSO trials demonstrate the potential of deucravacitinib to elevate the oral standard of care for individuals who are candidates for systemic therapy,” said Sadeh.

If approved on its PDUFA date of September 10, the drug would enter the market with only Otzelta as a competitor. BMS estimated that deucravacitinib has the potential to reach $4 billion sales if approved for multiple inflammatory disease indications. BMS is also expecting EMA approval later this year.

2. Mirati’s lung cancer drug

This past February, Mirati Therapeutics announced that the FDA had accepted its NDA for adagrasib, its drug candidate meant to treat patients with non-small cell lung cancer (NSCLC).

Adagrasib works by sustaining target inhibition, an attribute that could help treat cancers with KRAS mutation, as the protein regenerates every 24-28 hours. It’s being evaluated by itself and in combination with other cancer therapies for patients with not only KRAS G12C mutated solid tumors, but colorectal and pancreatic cancer as well.

Recently, Mirati reported that the disease control rate was 100% across a subset of 27 patients participating in the phase 2 KRYSTAL-1 study, which included patients suffering from gastrointestinal cancers.

Adagrasib is also being reviewed under the FDA’s Real-Time Oncology Review pilot program, which helps streamline revolutionizing treatments to get them to patients as early as possible. 

If approved on its PDUFA date of December 14, potential sales could reach $1.7 billion by 2026, giving adagrasib blockbuster status.

3. bluebird bio’s rare disease gene therapy

Bluebird bio’s beti-cel has the potential to not only be the first one-time gene therapy treatment for people with beta-thalassemia, but also the first ex vivo lentiviral vector gene therapy offered in the U.S.

Beta-thalassemia is a rare genetic blood disease caused by mutations in the beta-globin gene which can result in significantly reduced or absent adult hemoglobin production. Patients living with the most severe form often rely on blood transfusions for the duration of their lives, a process they have to undergo every 2-5 weeks.

Beti-cel is designed to add functional copies of a modified form of the B-globin gene into the patients’ hematopoietic stem cells. But the promise made by cell and gene therapies is complicated to keep.

“Gene therapies are complex, potentially transformative treatment options for those living with severe genetic diseases,” said Andrew Obenshain, CEO of bluebird, back in January when the company announced that the FDA had extended beti-cel’s review period.

**Update: On August 17, the U.S. FDA approved the drug — now branded as Zynteglo —  for adults and pediatric patients.

4. Roche’s Alzheimer’s disease treatment

Alzheimer’s drugs run the risk of not living up to their hype, with Aduhelm being the perfect example.

Roche’s gantenerumab, however, has garnered positive momentum after being put on the shelf for a few years. The company brought its phase 3 study back in 2018, with the hope that giving patients a higher dose would yield different results.

The drug is an IgG1 antibody developed to bind to aggregated forms of beta-amyloid and remove the plaques, which are believed to cause the neurodegenerative effects on the brain that characterize Alzheimer’s disease. Given as a subcutaneous injection, the drug has proven to lower amyloid plaque presence.

The number of Alzheimer’s cases is expected to rise to 78 million worldwide by the end of this decade. It’s the most common form of dementia, and effective treatments are scarce.

Gantenerumab received FDA Breakthrough Therapy designation in October 2021.

“This Breakthrough Therapy designation reinforces our confidence in gantenerumab, which would be the first subcutaneous medicine for the treatment of Alzheimer’s disease with the potential for at-home administration,” said Levi Garraway, Roche’s chief medical officer and Global Product Development head.

Gantenerumab is currently being investigated in eight clinical trials, including a phase 3 secondary prevention trial in participants at risk for or at the earliest stages of Alzheimer’s, and two phase 3 studies investigating gantenerumab versus placebo in participants who have early Alzheimer’s over 27 months. The latter two trials are nearing conclusion, which would allow Roche to seek FDA approval.

Gantenerumab is believed to have the potential to reach blockbuster status, with Evaluate Vantage estimating sales of $2.5 billion by 2026. 

About the Author

Andrea Corona | Senior Editor

Andrea Corona serves as the Senior Editor of Pharma Manufacturing — a leading source of news and insights for pharma professionals — and is responsible for creation of editorial content, moderating webinars, and co-hosting the "Off script" podcast. Her editorial journey started as an as associate editor at Biocompare, an online platform providing product information, industry news, articles, and other resources to support scientists in their work. Before Biocompare, she was a digital producer at Science Friday, focusing on adapting radio segments for the web and social media management. Andrea earned her bachelor's degree in journalism and biology from the State University of New York, at Purchase College.