In 2020, all goal dates met for CDER's novel approvals

Regulatory NewsRegulatory News | 13 January 2021 |  By 

Editor’s note: come back to Regulatory Focus tomorrow for an expanded examination of these approvals, including which sponsors made use of expedited programs, and which programs were used most frequently.
A slate of 53 drugs makes up the list of novel drugs approved by the US Food and Drug Administration in 2020. The list, released Wednesday, includes such notables as Veklury (remdesivir, Gilead), a shelved antiviral repurposed to treat COVID-19 infection, and a single monoclonal antibody plus a cocktail to treat Ebola virus infection.
FDA also gave the nod to Zokinvy (lonafarnib, Eiger), the first drug to treat some forms of premature aging, including progeria. Across all disease types, more than half of approvals (31/53, 58%) were for rare diseases, noted Patrizia Cavazzoni, MD, FDA’s acting director for the Center for Drug Evaluation and Research, in a post summarizing the year’s work.
According to Cavazzoni, all Prescription Drug User Fee Act (PDUFA) goal dates were met for the year’s novel approvals, and all but four of the approvals occurred on the first cycle. This 92% figure bests the average of 85% first-cycle wins for 2011-2019 novel drug approvals.
“New drug therapies for patients with rare diseases are often among the most important approvals. Patients with rare diseases frequently have few or no drugs available to treat their condition — and for them, approvals of so-called “orphan” drugs can mean new hope for an enhanced quality of life, and in some cases, increased survival,” wrote Cavazzoni.
Approximately a quarter of the novel drugs debuting in the US in 2020 are small molecules or targeted antibodies that received the nod to treat cancers, while novel therapies for neurological disorders such as multiple sclerosis, neuromyelitis optica spectrum disorder and migraine were also on the list.
Some very old drugs also appear on the novel approvals list: Artesunate (Amivas), approved to treat severe malaria, was developed in the 1970s. Lampit (nifurtimox, Bayer) first came into use in 1965. The medication was approved by FDA in 2020 to treat Chagas disease, and it appears on the World Health Organization’s List of Essential Medicines.
Other notables on the list include four radioisotopes meant for use in the detection of Alzheimer’s disease, prostate cancer and some neuroendocrine and breast cancers.
Vaccines, blood and blood products, plasma derivatives and cell and gene therapy products are excluded from the list.
New Drug Therapy Approvals Report


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