Since the advent of the COVID-19 virus, there have been diversions of a lot of biopharma resources into the race for treatment options and with the emergence of various variants, the effect of the virus on 2021 FDA approvals cannot be overlooked. A lot of PDUFA missed due to resources diversion, travel bans and many other factors. Despite these, the FDA approved 208 drugs in 2021, amongst these, there were 50 NMEs. This is in range with previous years with 53 approvals in 2020 and 48 in 2019.
Small molecules dominated the list again with 33 (66%) of the 50 approved NMEs in 2021. Monoclonal Antibodies (mAb) comprises 20% of all NMEs, accounting for most of the biologics, with the other 6 (12%) being proteins. Sarepta therapeutics’ casimersen was the only oligotherapeutics approved in 2021, increasing the number of therapies available for Duchenne Muscular Dystrophy (DMD).
The biopharmaceutical industry continues to benefit from advancements in chemistry, biology, biochemistry and other life sciences innovations. Drug discovery and development also continues to realize the benefits in the application of artificial intelligence (AI), particularly favoring the development of small molecule based therapies.
From the standpoint of new drug approvals, we continue to see traditional modalities, advanced therapies (e.g. cell and gene therapy) – have not produced a commensurate number of approvals despite hundreds of trials, dozens of companies and billions of investment dollars seen every year.
Biopharma companies have persevered in their search for cancer treatments despite COVID 19 diversions, the FDA approved 15 NMEs for the treatment of various oncology indications down from 17 NMEs in this therapy area approved in 2020, this was followed by 6 in rare diseases.
Companies like Merck KgaA, Rhizen Pharmaceuticals, Oncopeptides, Novartis, On Target Laboratories spearheaded Oncology NMEs.
Neurology had 6 NMEs, most of them being small molecules and 1 mAb. These include
Company | Drug | Indication |
Kempharm/Aquestive Therapeutics | Azstarys | ADHD |
Johnson & Johnson | Ponvory | Multple Sclerosis |
Supernus Pharmaceutical | Qelbree | ADHD |
Alkermes | Lybalvi | Schizophrenia |
Neurimmune/Biogen | Aduhelm | Alzheimer’s disease |
AbbVie | Qulipta | Episodic Migraines |
Rare Diseases had 6 NMEs for various indications including: Amondys 45 for Duchenne muscular dystrophy from Sarepta Therapeutics and Voxzogo for children with achondroplasia and open epiphyses from BioMarin Pharmaceutical.
The FDA also approved one imaging agent from Lantheus Holdings and one anti-infective from Scynexis which is one down from the two anti-infectives approved in 2020.
There were 16 partnered approved NMEs in 2021, this is less than the 25 partnered NMEs in 2020. 10 of which were small molecules, 5 mAbs and 1 protein:
Companies | Drug Name | Active Ingredient | Indications |
Bayer/Merck&Co. | Verquvo | vericiguat | Chronic Heart Failure |
Rhizen Pharmaceuticals/TG Therapeutics | Ukoniq | umbralisib | Lymphoma |
Orphatec/BridgeBio | Nulibry | fosdenopterin | Mineral Deficiency |
Kempharm/Aquestive Therapeutics | Azstarys | serdexmethylphenidate & dexmethylphenidate | ADHD |
Kyowa Kirin/Aveo Oncology | Fotivda | tivozanib | Renal Cell Cancer |
Supernus Pharmaceutical | Qelbree | viloxazine | ADHD |
Mayne Pharma/Mithra Pharmaceuticals SA | Nextstellis | drospirenone and estetrol tablets | Contraception |
AnaptysBio/GSK | Jemperli | dostarlimab-gxly | Endometrial Cancer |
Genmab/Johnson & Johnson | Rybrevant | amivantamab-vmjw | Non-Small Cell Lung Cancer |
BridgeBio Pharma/Novartis | Truseltiq | infigratinib | Cholangiocarcinoma |
Neurimmune/Biogen | Aduhelm | aducanumab | Alzheimer’s disease |
Sanofi | Fexinidazole | fexinidazole | Sleeping Sickness |
Cara Therapeutics/Vifor Pharma | Korsuva | difelikefalin | Pruritus |
Seagen Inc/Genmab A/S | Tivdak | tisotumab vedotin-tftv | Recurrent or Metastatic Cervical Cancer |
PharmaEssentia | Besremi | ropeginterferon alfa-2b-njft | Polycythemia Vera |
AstraZeneca/Amgen | Tezspire | tezepelumab-ekko | Severe Asthma |
In conclusion, recent years have seen continued dominance in small molecules and this is expected to continue as advances in technology make the drug discovery process more efficient compared to traditional approaches. While we are on the look for more non-traditional modalities, it still remains an open question if 2022 would be the year cell and gene therapy would begin to live up to its potential.
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