New Drug Approvals On Track at FDA

Despite the pressures and added regulatory challenges imposed by the COVID-19 pandemic, and the uproar over its surprise approval of Biogen’s new Alzheimer’s therapy Aduhelm, FDA is on track this year to authorize a notable number of new molecular entities (NMEs) and important biotech therapies. In the first half of 2021, the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) gave the nod to 29 novel therapies, slightly ahead of last year’s pace that saw the approval of a near-record 53 new drugs.

CDER’s list of novel new drug added up to 27 NMEs and biologics as of June 30, 2021, with several more additions in July.¹ AndCBER okayed a new pneumococcal vaccine and new treatments for multiple myeloma and for lymphoma.²

Almost half of the new drugs are treatments for cancer and related conditions, many benefitting from priority review and accelerated approval programs. That includes Jazz Pharmaceuticals’ new blood cancer therapy, a treatment for non-small cell lung cancer from Amgen, and Bristol Myers Squibb’s new CART-T therapy for multiple myeloma plus a cell therapy for large B-cell lymphoma.

Important non-cancer approvals also include the first new treatment for vaginal yeast infections in two decades and the first therapy for chronic weight management authorized since 2014, arising from approval of an added indication for Novo Nordisks’s type 2 diabetes injectable Wegovy (semaglutide) for obese or overweight adults. FDA also okayed new therapies for Duchenne muscular dystrophy, lupus nephritis, chronic heart failure and, notably, an extended release injectable to manage HIV, replacing daily pills with monthly injections.

There were advances in the over-the-counter drug world, too. In June, FDA approved the first nonprescription nasal antihistamine to expand treatment options for patients age six and older; younger children still have to obtain Bayer Healthcare’s Astepro (0.15% azelastine hydrochloride nasal spray) on a prescription basis.

Important news on the approvals front continued in July, as FDA authorized its first interchangeable biosimilar — Mylan’s Semglee once-daily insulin therapy, biosimilar to Sanofi’s Lantus.³ FDA also approved an important treatment for high-risk, early-stage, triple-negative breast cancer, giving the nod to Merck’s Keytruda (pembrolizumab) to be used in combination with chemotherapy for this serious condition.

On the downside, FDA halted use of a treatment approved in February for relapsed or refractory multiple melanoma, Pepaxto from the Swedish firm Oncopeptides, based on evidence from ongoing clinical studies of higher death rates among patients treated with the new drug vs. an earlier therapy. Pepaxto gained accelerated approval, but now FDA has alerted patients and health professionals to the potential risk and has suspended enrollment in postapproval studies pending further examination of the safety signal.⁴

CDER officials attribute their ability to maintain a fairly normal advise and consent process for innovative drugs over the past 18 months on the hard work of its dedicated staff, as well as efficiencies gained from the recent reorganization of its Office of New Drugs (OND). CDER director Patrizia Cavazzoni noted increases in staff hiring and retention at the May 2021 annual meeting of the Food and Drug Law Institute (FDLI), attributing this surprising development to new remote work flexibilities due to COVID-19 restrictions. She also cited the success of large platform trials and clinical networks, as well as increased sponsor use of decentralized trials. The structural overhaul of OND has resulted in smaller, more flexible review offices with clearer areas of expertise and greater alignment of interrelated disease areas, Cavazzoni explained in recent testimony to the House Energy & Commerce Health subcommittee, which held a hearing to explore the lack of new therapies for neurogenerative diseases.⁵

Notes

1. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2021

2. https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2021-biological-license-application-approvals

3. https://www.fda.gov/news-events/press-announcements/fda-approves-first-interchangeable-biosimilar-insulin-product-treatment-diabetes?utm_medium=email&utm_source=govdelivery

4. https://www.fda.gov/drugs/drug-safety-and-availability/fda-alerts-patients-and-health-care-professionals-about-clinical-trial-results-showing-increased?utm_medium=email&utm_source=govdelivery

5. https://www.fda.gov/news-events/congressional-testimony/path-forward-advancing-treatments-and-cures-neurodegenerative-diseases-07292021