Another year of firsts in FDA’s new drug approvals
Targeted therapies beat out immuno-oncology, new modalities in 2020’s crop of new drug approvals
The pandemic didn’t slow drug approvals in the U.S., but the market is still waiting for new modalities and the next wave of immuno-oncology therapies.
By Lauren Martz, Senior Editor
January 1, 2021 2:57 AM UTC
The COVID-19 pandemic didn’t stand in the way of new drug approvals by FDA in 2020, but few new modalities crossed the finish line despite the clinical development fervor around platform technologies and new drug constructs.
FDA’s Center for Drug Evaluation and Research (CDER) approved 53 new drugs, surpassing 2019’s count of 48. The 2020 count ties with 1996 for the second-most approvals ever behind 2018’s 59 new drugs.
An additional eight novel biologics were approved by the Center for Biologics Evaluation and Research (CBER) in 2020.
Those drugs made it through the regulatory process despite the COVID-19 pandemic’s global impact. During the first half of the year, many operations were halted and biopharma industry executives were concerned over potential delays in regulatory reviews, but the coronavirus doesn’t seem to have slowed FDA.
Nearly half (49%) of the 53 approvals by CDER happened during the first half of the year, as did seven of the eight (88%) approvals by CBER.
Despite the challenges, 2020 was a year of firsts in new drug approvals, continuing a trend apparent in 2019’s new drug class.
Among the 2020 approvals are nine drugs that hit novel targets not addressed by other approved therapies.
The most notable may be Blenrep belantamab mafodotin-blmf, an antibody-drug conjugate against BCMA from GlaxoSmithKline plc (LSE:GSK; NYSE:GSK), which was approved in August. Blenrep beat out a series of bispecific antibodies and CAR T cell therapies against the target in one of the most closely watched races to the market in oncology.
The other antibody-drug conjugate to gain approval in the U.S. also hits a hot new oncology target. The TROP2-targeting ADC Trodelvy sacituzumab govitecan-hziy from Gilead Sciences Inc. (NASDAQ:GILD) was approved in April to treat metastatic triple-negative breast cancer.
A clinical and preclinical pipeline of at least four other therapies against the target, with modalities including antibodies and cell therapies, follows Trodelvy.
The year also produced some of the first selective inhibitors of several kinases as precision oncology emerged as a big trend in the approvals.
Examples include Tabrecta capmatinib from Incyte Corp. (NASDAQ:INCY) and Retevmo selpercatinib from Eli Lilly and Co. Inc. (NYSE:LLY), which were approved two days apart in May. Tabrecta is the first selective c-Met inhibitor approved to treat non-small cell lung cancer (NSCLC) patients with a mutation in c-Met that leads to exon 14 skipping. Retevmo was the first selective Ret inhibitor approved in the U.S.
Six therapies in the new approval group were the first ever approved for their indications.
2020 saw the first COVID-19 therapeutic approval in Gilead’s Veklury remdesivir. Veklury gained full approval in October after receiving emergency use authorization in May.
The first therapeutic for Ebola virus — Inmazeb atoltivimab, maftivimab and odesivimab-ebgn from Regeneron Pharmaceuticals Inc. (NASDAQ:REGN)— was approved in October. It follows the first vaccine approval for the indication in 2019.
The breakout star among indication firsts, in terms of launch performance, is Tepezza teprotumumab-trbw from Genmab A/S (CSE:GMAB; NASDAQ:GMAB) and Horizon Therapeutics plc (NASDAQ:HZNP). The anti-IGF-1 mAb was approved to treat thyroid eye disease and has overwhelmingly exceeded Horizon’s initial guidance of $30-40 million. In November, the company increased its 2020 sales guidance to $800 million from $650 million.
The first therapy approved for peanut allergy, Palforzia from Aimmune Therapeutics Inc., faced more COVID-related launch challenges before Nestlé Health Sciences announced its plans to acquire the company for $2.6 billion in August.
Missing from the list
The list of new drug approvals in 2020 highlights plenty of firsts, but doesn’t reflect the industry’s level of commitment to immuno-oncology and new modalities.
In immuno-oncology, first-generation checkpoint inhibitors and CAR T cell therapies continue to receive broad label expansions, but very few new drugs have reached the market since the initial wave of approvals at least three years ago.
The one exception this year is Tecartus brexucabtagene autoleucel, a CD19-targeted CAR T cell from the Kite Pharma Inc. unit of Gilead that was approved to treat relapsed or refractory mantle cell lymphoma in July.
No other immuno-oncology therapies reached the market in the U.S. this year.
Beyond Kite’s CAR T and the two antibody-drug conjugates, only two other new modality therapies are present in the 2020 crop of approvals, even though several new modalities continued to make huge clinical strides in 2020 and were the primary focus of major scientific meetings.
FDA approved siRNA therapy Oxlumo lumasiran from Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) and antisense oligonucleotide Viltepso viltolarsen from Nippon Shinyaku Co. Ltd. (Tokyo:4516).
Notably absent from the list of approvals are two therapeutic modalities with mounting clinical and regulatory validation: bispecific antibodies and gene therapies.
Bispecific antibodies have attracted more oncology deals than any other modality over the past three years and are gaining traction among pharmas and biotechs for their ease of manufacture and safety advantages over CAR T cells.
But it’s been nearly three years since Blincyto blinatumomab from Amgen Inc. (NASDAQ:AMGN) was approved as the first bispecific T cell engaging antibody, and the market is still waiting for the next-generation drugs.
Gene therapies have been trickling onto the market over the past three years. Last year’s approval of Zolgensma onasemnogene abeparvovec-xioi for spinal muscular atrophy (SMA) was a major advancement for patients, but there hasn’t been a flood of new gene therapy approvals.
TARGETS BCMA (TNFRSF17; CD269) – Tumor necrosis factor receptor superfamily member 17 c-Met (MET; HGFR) – c-Met receptor tyrosine kinase IGF-1 – Insulin-like growth factor 1 RET – Ret proto-oncogene TROP2 (TACSTD2; EGP-1) – Tumor-associated calcium signal transducer 2
The COVID-19 pandemic didn’t stand in the way of new drug approvals by FDA in 2020, but few new modalities crossed the finish line despite the clinical development fervor around platform technologies and new drug constructs.
FDA’s Center for Drug Evaluation and Research (CDER) approved 53 new drugs, surpassing 2019’s count of 48. The 2020 count ties with 1996 for the second-most approvals ever behind 2018’s 59 new drugs. ...
