On January 2, 2020, the FDA released the CDER annual report, stating that 2019 was “another strong year for innovation and advances.” The FDA cleared 48 new drugs for market, making it the second most productive year in the last decade (2018 approved 59 drugs). But given the fact that the total number of submissions have gone down, and that the American government was shut down for a lengthy period at the beginning of the year, the FDA delivered an impressive result in 2019.
I’d like to share some of my personal thoughts
after reading this report. Here are a few notable highlights and my takeaways:
1. 21 of CDER’s 48 novel drug approvals, or 44%, were orphan drugs
What does that tell us?
* As Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, puts it, “New drug therapies for patients suffering from 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, survival.”
* Patient unmet needs and market value merges where the pharmaceutical companies pay attention and take practical approaches. One of the most promising avenues that big pharmas can take is to partner with non-profits focused on particular unmet needs, and to collaborate with small biotech companies that are specializing in tackling unmet needs. One such example is Beacon Discovery, which has partnered with several leading companies, focusing on helping them with early-stage research and discovery.
If 2019 was a good year for orphan drugs, 2020 is already getting off to a great start. In Q1, three more possible orphan drugs to be on the look-out for include: Oxbryta, the first treatment to address the root cause of sickle cell disease (SCD); Adakveo, an injectable treatment for SCD patients who suffer from vaso-occlusive crises (VOC); and Givlaari, a drug to treat the blood disorder acute hepatic porphyria (AHP). Learn more about these drugs here.
Some of these new drugs in development are built
on very exciting new innovations:
* The above-mentioned Givlaari is based on RNA interference (RNAi), a technique that silences abnormal gene expression. As BioSpace reports, Givlaari is only the second drug to be approved leveraging RNAi.
* The cancer drug Rozlytrek, for pan-tumor use, marks only the third time that the FDA has “cleared a cancer drug to treat tumors based on a specific genetic signature rather than by location in the body,” explains BioPharmaDive.
As evidenced by the emergence of innovative drugs like these, my personal opinion is that we are truly transitioning into the era of precision medicine and personalized medicine, when more and more oncology drugs are being developed based on the use of biomarkers and targeted delivery of medicine.
3. 10 new biosimilars
Biosimilars, which are drugs that are very
similar to already-approved biologics, had a busy regulatory year in 2019, with
many new approvals and new guidance documents published.
There were 10 biosimilar approvals covering
indications for numerous conditions, such as rheumatoid arthritis, plaque
psoriasis, breast cancer, metastatic stomach cancer, metastatic colorectal
cancer, non-squamous non-small cell lung cancer, glioblastoma, metastatic renal
cell carcinoma, cervical cancer, B-cell non-Hodgkin’s lymphoma, chronic
lymphocytic leukemia, granulomatosis with polyangiitis, and microscopic
“Biosimilars have great potential for both patients and the entire health care system. As patents and exclusivity protections for biologics expire in the United States, we can expect many more biosimilars to be submitted for approval. More products on the market means greater competition that can lead to increased access to therapies and lower costs to patients.”
– Janet Woodcock
4. The first-ever FDA novel approval drug from a Chinese pharmaceutical company
November 14, 2019 was a clear milestone for Chinese pharmaceutical companies, as it was then that Brukinsa from BeiGene received FDA approval for the very first novel therapy from China in history. Regulatory reform in China has enhanced innovative drug discovery and clinical development, encouraging innovation and speeding patient access to new drugs.
Studies showed that BeiGene’s drug has real promise. “The FDA’s decision is largely based on tumor shrinkage data from a phase 2 trial in 86 Chinese patients,” reports FiercePharma. “In that single-arm study, Brukinsa triggered an overall response rate of 84% with a median duration of response of 19.5 months. Some 59% of patients saw a complete response, meaning their cancers were undetectable after treatment.”
A mission to work together
To quote Janet Woodcock once more: “CDER’s mission goes well beyond
critically reviewing the safety and efficacy of drug applications we receive
from industry … We are working to develop more innovative and efficient
approaches for the development and study of the drug therapies that will emerge
from these technological advances.”
I believe this is also the dream and the
mission of most people working in the pharmaceutical industry: to work
collaboratively with partners across academia, industry, patients, caregivers
and regulators to provide true benefits for patients. We at Elsevier Life
Sciences solutions all share that same mission.
If you are interested in finding out more about collaborations to support drug discovery and development, please read more on our website.