FDA decisions to watch

March 31, 2022
The U.S. regulator will soon have to make the call on a handful of highly-anticipated treatments

The U.S. Food and Drug Administration has a series of important decisions to make in upcoming months, with everything from potential blockbuster launches to pandemic guidance hanging in the balance.

New BMS heart drug

When Bristol Myers Squibb shelled out $13.1 billion to buy MyoKardia in late 2020, mavacamten, MyoKardia’s lead asset, was a driving force behind the deal. BMS believed that the oral myosin inhibitor had big potential as a first-in-class therapy that could address the underlying cause of obstructive hypertrophic cardiomyopathy (HCM).

At the time of the MyoKardia deal, BMS CEO Giovanni Caforio raved on CNBC that the drug was a  “potentially revolutionary medicine for the treatment of the very serious disease.”

Obstructive HCM is a chronic disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can make it difficult for blood to circulate to the rest of the body — leading to debilitating symptoms and cardiac dysfunction.

This past Nov., the FDA pushed the PDUFA date for mavacamten back three months to April 28, 2022, to allow sufficient time to review updates to the proposed Risk Evaluation Mitigation Strategy. But BMS is eager to offset the fast-approaching Revlimid patent cliff and still has its eye on the mavacamten prize. During its recent presentation at J.P. Morgan Health Care Conference, the drugmaker tagged mavacamten as a $4 billion opportunity.

Eli Lilly’s diabetes/obesity treatment

With Biogen’s struggles wounding hopes for Eli Lilly’s Alzheimer’s candidate, donanemab, more attention has shifted to tirzepatide. Intended as a treatment for patients with type 2 diabetes or obesity, tirzepatide has demonstrated the ability to improve blood sugar levels and weight loss in clinical trials.

If approved, the GLP-1/GIP dual agonist will compete with Novo Nordisk’s Ozempic and Sanofi’s Lantus. All three therapies use insulin glargine as the active ingredient. Lilly plans to use a priority review voucher and expects tirzepatide to be approved around mid-year in the U.S., EU and Japan. 

Eli Lilly CFO, Anat Ashkenazi, expects to hit the ground running and says the company will waste no time getting the drug to market. “As we’ve indicated, we expect to see an approval followed by a rapid launch right after that. We’d typically launch within days to weeks after approval,” said Ashkenazi during the Cowen 42nd Annual Healthcare Conference in early March.

The rush to market is no surprise as tirzepatide has big commercial potential. In its 2022 Preview report, Evaluate Vantage estimated sales could reach in $5.4 billion in 2026.

The drug’s pricetag, however, is still up for debate. The Institute for Clinical and Economic Review, a pricing watchdog whose reports are often used by drug manufacturers  and payers alike, recently gave the Lilly drug a middle-of-the-road C+ grade on comparable health benefits, citing lack of data about cardiovascular and renal outcomes control. Based on this, ICER said the treatment should have an annual price of between $5,500 and $5,700. Ashkenazi said Lilly will focus on demonstrating the value of the drug from a weight loss and A1C level controls perspective while negotiating with payers.

EUAs for 4th COVD vaccines doses

With the emergence of new and more contagious variants, many Americans have been left wondering how to proceed with additional COVID-19 vaccine boosters. In mid-March, Pfizer and BioNTech, followed by Moderna, submitted applications to the FDA for emergency use authorization of an additional booster dose of their respective vaccines.

Moderna also recently announced that it is preparing to submit data for initial COVID-19 boosters in the 12-17 years age group. Pfizer already has authorization for a single initial booster dose for those 12 years and older.

In late March, Pfizer announced that the FDA expanded the emergency use of the Pfizer-BioNTech vaccine to include a second booster dose for adults ages 50 years and older as well as for individuals 12 years and older who have been determined to have certain kinds of immunocompromise. At the same time, a second booster dose of the Moderna COVID-19 vaccine was authorized in individuals 18 years and older with the same kinds of immunocompromise.

Both the Pfizer and Moderna 4th dose submissions were based in part on recently published data from a 700-person trial generated in Israel following the emergence of omicron. Israel began offering fourth shots to test groups of medical workers back in December and is now gradually broadening eligibility.

The FDA had said the agency will review the EUA requests “as expeditiously as possible,” but did not indicate decision dates. 

While the expanded EUAs for additional boosters for specific patient groups came quicker than anticipated, the FDA still has decisions to make regarding additional boosters for the rest of the population. The agency announced that it will convene its independent Vaccines and Related Biological Products Advisory Committee virtually on April 6 to “discuss considerations for future COVID-19 vaccine booster doses and the strain selection process to address current and emerging variants during the Fall season 2022,” however, no vote is planned for the meeting and the agency said there will not be discussion of product-specific applications. 

About the Author

Karen P. Langhauser | Chief Content Director, Pharma Manufacturing

Karen currently serves as Pharma Manufacturing's chief content director.

Now having dedicated her entire career to b2b journalism, Karen got her start writing for Food Manufacturing magazine. She made the decision to trade food for drugs in 2013, when she joined Putman Media as the digital content manager for Pharma Manufacturing, later taking the helm on the brand in 2016.

As an award-winning journalist with 20+ years experience writing in the manufacturing space, Karen passionately believes that b2b content does not have to suck. As the content director, her ongoing mission has been to keep Pharma Manufacturing's editorial look, tone and content fresh and accessible.

Karen graduated with honors from Bucknell University, where she majored in English and played Division 1 softball for the Bison. Happily living in NJ's famed Asbury Park, Karen is a retired Garden State Rollergirl, known to the roller derby community as the 'Predator-in-Chief.'