2018 was record year for FDA drug approvals

Pharma Plethora

The US Food and Drug Administration (FDA) approved an all-time high number of drugs in 2018. Of the 59 new molecular entities that obtained the FDA’s approval to go to market, there was an assortment of small molecules, biologics, and new modalities, according to an article by Lisa M. Jarvis in Chemical & Engineering News.

The largest share of new drugs came in the form of small molecules, accounting for 64 percent of the therapies FDA approved in 2018. Antibodies accounted for 20 percent of the FDA approvals. Fully 15 percent of 2018’s newly approved drugs fell outside of the two main modalities. Last year, very diverse approaches were taken by researchers to conquer diseases, especially rare genetic diseases.

Jarvis opined that one of the “highlights of the year” was the approval of the first drug, Alnylam Pharmaceuticals’ Onpattro, that acts by means of RNA interference. As she explained, “Onpattro uses short, double-stranded nucleotides to intercept mRNA before it can deliver the instructions for assembly of an unwanted protein.” First approved August 10, 2018, Onpattro (patisiran) is a small interfering ribonucleic acid (siRNA) therapeutic for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR).

While that approval was a big scientific achievement, the commercial viability of the product and RNAi technology as a whole is still a question. A mere two months after Onpattro got onto the market, Akcea Therapeutics and Ionis Pharmaceuticals received FDA approval for an antisense oligonucleotide for the same rare disease addressed by Onpattro. Pfizer has been working on a small molecule to treat the disease for a long time.

Jarvis described Loxo Oncology and Bayer’s Vitrakvi as “another notable new drug” that became the second cancer therapy to be approved to treat people based on their genetic profile, rather than the location of their tumor. In toto, the FDA approved 16 new cancer therapies, which represented an increase from the 12 cancer drugs given the nod by the FDA in 2017. Of the oncology drugs that received approval, seven are designed to treat some type of blood cancer.

Cancer drugs approved were Copiktra (duvelisib) from Verastem for the treatment of chronic lymphocytic leukemia, small lymphocytic lymphoma or follicular lymphoma; Daurismo (glasdegib) from Pfizer for treating newly-diagnosed acute myeloid leukemia in adults 75 years of age or older; Erleada (apalutamide) from Janssen Oncology for treating prostate cancer; Libtayo (cemiplimab-rwlc) from Regeneron Pharmaceuticals for treating cutaneous squamous cell carcinoma; Lorbrena (lorlatinib) from Pfizer for treating ALK-positive metastatic non-small cell lung cancer; Lumoxiti (moxetumomab pasudotox-tdf) from AstraZeneca for treating relapsed or refractory hairy cell leukemia; Lutathera (lutetium Lu 177 dotatate) from Advanced Accelerator Applications for treating gastroenteropancreatic neuroendocrine tumors; Poteligeo (mogamulizumab-kpkc) from Kyowa Kirin for treating mycosis fungoides or Sézary syndrome; Talzenna (talazoparib) from Pfizer for treating deleterious germline BRCA-mutated HER2-negative locally advanced or metastatic breast cancer; Tibsovo (ivosidenib) from Agios Pharmaceuticals for treating acute myeloid leukemia with a susceptible IDH1 mutation; Vitrakvi (larotrectinib) from Loxo Oncology for treating solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion; Vizimpro (dacomitinib) from Pfizer for treating metastatic non-small cell lung cancer; and Xospata (gilteritinib) from Astellas for treating acute myeloid leukemia with a FLT3 mutation.

In addition to new modalities and cancer drugs, last year’s FDA approvals covered treatments for long-underserved diseases. They included a new class of drug for migraine prevention, the first new pill for endometriosis in more than a decade and the first marijuana-extracted treatment, Epidiolex, for a rare form of epilepsy.