The U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) had a busy 2021.
The agency approved 50 novel drugs and several new biosimilar drugs — including the first two interchangeable biosimilars — according to an annual FDA report.
Orphan drug approvals made a big leap in 2021, with 52% of CDER-approved drugs falling into the designation over the past year. Included in these approvals were: BioMarin Pharmaceutical’s Voxzogo, which was approved in November as a way to increase height in people with achondroplasia — the most common type of dwarfism; Sanofi's Nexviazyme, an infusion to treat patients one year and older with late-onset Pompe disease; and Sarepta Therapeutics’ Amondys 45, an injection for the treatment of Duchenne muscular dystrophy.
The number of orphan drug approvals has been rising over the past years, although numbers have decreased slightly from the 31 orphan drug approvals in 2020. The passage of the Orphan Drug Act in 1983, which provides financial incentives for pharmaceutical companies to develop drugs that have a much smaller consumer base than other treatments, has led to an increase in the number of orphan drugs in development.
First-in-class drugs also made a big splash in 2021, with 54% of novel drugs approved as first-in-class. This drug category has a different mechanism of action from existing therapies, meaning they act differently in the body than other drugs do. Key players in this category include LEO Pharma’s Adbry — a monoclonal antibody treatment for the treatment of moderate to severe eczema — which was approved in December.
In an FDA first, two interchangeable biosimilar treatments — Mylan Pharmaceuticals’ Semglee and Boehringer Ingelheim’s Cyltezo — were approved. Similar to how generic drugs can be substituted for brand-name drugs, interchangeable biosimilars can be substituted for the reference product without prescriber intervention. Semglee was approved by the FDA on July 28 as a biosimilar for Sanofi's Lantus insulin. Cyltezo was approved as the first biosimilar to AbbVie's mega-blockbuster, Humira, treat a variety of conditions like Crohn’s disease, ulcerative colitis and different types of arthritis.
The report included some of 2021’s most memorable drugs, like Biogen’s Alzheimer’s drug Aduhelm. Aduhelm has been the subject of controversy over inconsistent results, high costs and dangerous side effects, including reports of stroke. Aduhelm received Fast Track, Priority Review, Accelerated Approval and first-in-class designations.
Another highly anticipated approval from 2021 was Viiv's Cabenuva, the first extended-release injectable drug meant to prevent HIV-1 infection in adults and adolescents.
CDER: Speeding drug approvals
The report also highlighted CDER’s efforts to improve the speed at which it can safely approve drugs. The center reported that 49 out of 50 novel drugs were approved by their originally scheduled target date and that 38 out of 50 novel drugs were approved in the U.S. before any other country. With four pathways to expedited approval, CDER is focusing on getting drugs approved faster than ever.
CDER has options when it comes to getting new therapies to patients. Drugs may be classified in four different ways to receive expedited approval. Fast Track Approval speeds development by increasing communication between FDA and drug developers and enabling CDER to evaluate portions of drug applications on a rolling basis, according to the agency.
In 2021, 18 out of the 50 novel drugs approved received Fast Track Designation. Breakthrough Therapy designation offers the same features as the Fast Track Designation but allows for more intensive FDA guidance. Fourteen out of the 50 novel drugs received Breakthrough Therapy designation.
If CDER determines a drug has the potential to provide significant advances in medical care, it can receive Priority Review designation. In 2021, 34 out of 50 novel drugs received Priority Review designation. The final path to expedited approval a drug can receive from CDER is Accelerated Approval. Under this designation, the center aims to bring drugs to market quicker than traditionally possible by providing more flexibility in approval endpoints.
Fourteen out of 50 novel drugs were approved in 2021 under Accelerated Approval. Of the 50 treatments, 37 received multiple expedited designations in 2021, including Novartis’ Scemblix, which received all four expedited approval designations and was approved by the FDA in November to treat chronic myeloid leukemia.
“I enjoy working with so many talented individuals in CDER who are determined to bring new drug therapies to patients as quickly as possible, while also ensuring that every approval is based on our consistently high standards for science, safety and effectiveness,” said Patrizia Cavazzoni, director of CDER, in an introduction to the report.