FDA Advances New Drugs, Biosimilars

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.

Almost half of the new drugs are treatments for cancer and related conditions, notably a new therapy for high-risk, early-stage, triple-negative breast cancer. Among important non-cancer approvals are the first new treatment for vaginal yeast infections in two decades and the first therapy for chronic weight management since 2014. FDA also okayed new therapies for Duchenne muscular dystrophy, lupus nephritis, chronic heart failure, plus an extended release injectable to manage HIV, replacing daily pills with monthly injections. There were advances in the over-the-counter drug world, too, as FDA approved the first nonprescription nasal antihistamine for pediatric patients age six and older.

CDER officials attribute their ability to maintain a fairly normal advise and consent process for innovative drugs 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), possibly due to remote work flexibilities during the pandemic, as well as the success of large platform trials and clinical networks and 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 said.

Questioning accelerated approval

Meanwhile, CDER’s surprise approval of Biogen’s Aduhelm has generated outrage, focusing attention on FDA efforts to make promising new therapies available to seriously ill patients based on early clinical results. Richard Pazdur, director of FDA’s Oncology Center of Excellence (OCE), rose to the agency’s defense, noting that accelerated approval has brought many important treatments to patients years before the completion of confirmatory studies, and that relatively few such products have been removed from the market post-approval, at a discussion in July sponsored by Friends of Cancer Research (FOCR). Instead of attacking the program, we need to address “what’s going right” with it, Pazdur said. He cited 165 accelerated approvals over the last 10 years, with only about 10 drugs removed from the market. “If you just look at the problems, you miss the broad gains” of the process, he said.

Aduhelm’ s high price, however, has accelerated the criticism. The HHS inspector general has agreed to investigate FDA’s decision-making process related to this approval, Congressional committees are planning to hold hearings on the program, and Medicare has launched a process to weigh coverage of the drug. It remains to be seen if FDA’s action accelerates the development of additional Alzheimer’s treatments or generates ongoing criticism of the agency’s approval process.

More biosimilars

Another important FDA approval in July is for the first interchangeable biosimilar—Semglee insulin therapy, made by Viatris and Biocon Biologics (formerly Mylan), interchangeable to Sanofi’s Lantus, a long-acting insulin analog. Since Congress authorized biosimilar development in 2010, FDA has approved only 30 follow-on biologics, and uptake by physicians and patients has been disappointing. Extensive patent protections and complex legal issues have enabled innovators to delay new competition, as has the need for a physician to prescribe the product.

Now all eyes are watching closely to see how well an interchangeable product competes with a well-established brand. FDA Acting Commissioner Janet Woodcock praised the action as “momentous,” emphasizing the agency’s support for a competitive marketplace for biological products and for increasing patient access to “safe, effective, and high-quality medications at potentially lower cost.”

It is no surprise that the first interchangeable is for an insulin therapy with potential to reduce costs for some of the nation’s 34 million diabetics and setting the stage for what analysts expect to be a surge in the development of interchangeable products.

Jill Wechsler is Pharm Exec’s Washington Correspondent. She can be reached at jillwechsler7@gmail.com.