FDA Watch: Approvals to watch for in 2021

By Charlie Williams
Published December 18, 2020

Key Takeaways

2020 may forever be remembered as the year of the coronavirus, but if you’re on the hunt for silver linings, here’s a big one: This chaotic year is almost over—and thanks to a few new vaccines, we can only hope the pandemic might soon be a thing of the past, too.

Therein lies the power of the FDA. It can vet and approve drugs like the new coronavirus vaccines that can save hundreds of thousands of people from sickness and death.

Despite the myriad challenges of the pandemic, researchers and government scientists at the FDA spent much of 2020 laying the foundation for a healthier, happier 2021—a year when the agency is poised to review and potentially approve more than 50 drugs in just the first 6 months.

Here are eight tentative approvals to watch for in 2021. 

Vericiguat for heart failure

This drug, which comes from the joint labs of Merck and Bayer, heads toward potential approval based on mixed data. Vericiguat was no better than placebo at reducing deaths in a phase 3 trial, but that same trial linked the drug to statistically significant reductions in a composite measure of cardiovascular deaths and heart failure hospitalization, which helped it hit its primary endpoint. 

Despite this, vericiguat, a soluble guanylate cyclase stimulator, garnered enough interest at the FDA to earn priority review in July, with a Prescription Drug User Fee Act (PDUFA) date set for January 20, 2021.

Trilaciclib for small cell lung cancer

The FDA granted trilaciclib priority review and accepted its New Drug Application (NDA) in mid-August. The drug is a first-in-class investigational therapy for the treatment of patients with small cell lung cancer, designed to preserve bone marrow and immune system function during chemotherapy. 

Currently, there aren’t any therapies to protect patients from chemotherapy-induced toxicities before they occur. If trilaciclib is approved after its PDUFA date on February 15, 2021, it would be the first proactively administered myelopreservation therapy intended to make chemotherapy safer and reduce the need for rescue interventions.

Oral paclitaxel and encequidar for metastatic breast cancer

Manufactured by Athenex Oncology, oral paclitaxel and encequidar secured the FDA’s acceptance for its NDA filing this past fall. This was supported by data from a phase 3 randomized, controlled clinical trial, which achieved its primary endpoint, showing statistically significant improvement in overall response rate and lower neuropathy for oral paclitaxel and encequidar vs intravenous paclitaxel. 

If approved, it would represent an alternative to IV infusions and allow patients to take the therapy in their homes instead of in the clinic—of particular importance during the coronavirus pandemic. The drug’s PDUFA date is February 28, 2021.

Aducanumab for Alzheimer disease

The FDA hasn’t approved a new therapy for the treatment of Alzheimer disease since 2003, notes the New York Times, but that could change in 2021. All eyes are on aducanumab, the Biogen drug that would be the first treatment to reduce the clinical decline of Alzheimer disease and become the first therapy to demonstrate that removing amyloid beta results in better clinical outcomes, if approved on its PDUFA date of March 7, 2021. 

Approval is an open question, though, according to the Times article. A federal panel of medical experts rejected arguments in support of aducanumab in November 2020, claiming the evidence wasn’t persuasive enough to warrant approval. The panel’s opinion is nonbinding, but the FDA has historically voted in support of their advisory committees in the past. 

Pimavanserin for dementia-related psychosis

Set for a PDUFA target date of April 3, 2021, Acadia Pharmaceuticals’ pimavanserin would be the first therapy indicated for the treatment of hallucinations and delusions associated with dementia-related psychosis, if approved. To date, the FDA has not identified any potential review issues during evaluation and does not plan to hold an advisory committee meeting. 

The drug’s supplemental NDA is supported by data from the phase 3 HARMONY trial, which met its primary endpoint, demonstrating that pimavanserin significantly reduced the risk of relapse of psychosis by 2.8 fold compared to placebo. 

Tenapanor for chronic kidney disease

On September 15, 2020, the FDA accepted Ardelyx Inc.’s NDA of tenapanor for controlling serum phosphorus in adult patients with chronic kidney disease on dialysis. The FDA has set a PDUFA target date of April 29, 2021.

The drug’s application is supported by three phase 3 trials of more than 1,000 patients. After about 20 months of treatment with tenapanor alone or in combination with low doses of sevelamer, patients exhibited a mean serum phosphorus reduction of 2.33 mg/dL.

Relugolix combination tablet for uterine fibroids

Most women will develop one or more uterine fibroids during their reproductive years. If approved, oral relugolix combination tablet, manufactured by Myovant Sciences, could help these women manage heavy menstrual bleeding associated with the condition. 

The drug’s target action date is June 1, 2021, after which it could become an alternative to invasive surgery for the estimated 5 million women who suffer from uterine fibroids in the United States. 

Rimegepant for migraine

The FDA accepted Biohaven Pharmaceuticals’ supplemental NDA for rimegepant in mid-October and has set a target action date for June 14, 2021. Approval would make it the first CGRP-targeting agent with regulatory approval as a single medication to treat acute episodes and prevent future migraine attacks.

In placebo-controlled trials, orally disintegrating rimegepant tablets have been shown to clear migraine symptoms within 60 minutes. They’ve also been shown to reduce monthly migraine days at the same 75 mg dose administered every other day for preventive treatment.

Share with emailShare to FacebookShare to LinkedInShare to Twitter