While FDA’s overall new drug approvals dropped in 2019, small molecular drugs continue to lead the way in novel approvals.
“Smaller molecules are more likely to be designated as first in class or breakthrough therapies by the FDA, compared to [other molecules] that aren’t structurally novel,” Todd Wills, managing director at CAS, which collects and analyzes the world’s disclosed science, told FormularyWatch.
Compared to 59 new drug approvals in 2018, FDA approved 48 new drugs (not biologics), FDA’s Center for Drug Evaluation and Research said in a recent report.
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Small molecules are still the primary modality for new drugs, according to Wills, accounting for 70% of the drugs approved in 2019. Seventy-four percent of those drugs were structurally novel (based on new scaffolds).
The top 3 innovative small molecule approvals in 2019, according to Wills are:
1. Rozlytrek (entrectinib): “This is the third time the agency has approved a cancer treatment based on a common biomarker across different types of tumors rather than the location in the body where the tumor originated,” Wills says.
2. Balversa (erdafitinib): This is first personalized treatment targeting susceptible FGFR genetic alterations for patients with metastatic bladder cancer, Wills notes.
3. Mayzent (siponimod): the first oral drug specifically for patients with active secondary progressive multiple sclerosis (SPMS).
Center for Drug Evaluation and Research (CDER) noted FDA approved several innovative drugs in 2019, including:
- Advances for certain patients with prostate cancer, bladder cancer, breast cancer, and lung cancer.
“We also approved two new bone marrow cancer therapies,” CDER says.
- Two new treatments for certain adult patients with multiple sclerosis.
- A new treatment for adults with depression who have tried other medications without success.
- A monoclonal antibody for the treatment of episodic cluster headache, “an extremely painful and often debilitating condition,” according to the CDER.
- A new drug to treat patients with Parkinson's disease (PD) who experience “off” episodes, during which medications are not working well.
- Two new drugs to help patients who suffer from migraine.
- Two notable treatments for cystic fibrosis (CF): 1.) the first triple combination therapy to treat patients with CF who have the most common form of the CF gene mutation and 2.) a new use for an already FDA-approved CF drug to treat certain patients as young as 6 years of age, which previously was approved only for certain patients 12 years of age or older.
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