2017 was a multi-blossoming year for global biopharmaceutical R&D, with several new drug development milestones. From technology innovation and R&D pipeline to clinical approval and marketing, 2017 can be described as a multi-point blossoming year for global new drug R&D, which also gives higher expectations for the new drug market.
In 2017, the U.S. Food and Drug Administration (FDA) approved 46 new drugs, 35 of which were approved for the first time in the world; the EU approved 51 new drugs, four of which were approved for the first time in the world; and Japan also approved 23 new drugs, three of which were approved for the first time in the world. A total of 120 new drugs were approved globally in Europe, the US and Japan.
More than 40 to 50 new drugs are expected to be marketed in 2018, including those in the review stage and those scheduled to be submitted for marketing in the first half of this year. According to the “New Drug Review Report” released by the Center for Drug Evaluation and Research (CDER) in December 2017, new drugs in the review stage will lay the foundation for the first marketing applications to be submitted in the EU, Japan, China, etc.
2017 Global Pharmaceutical R&D Harvest Year
In 2017, the U.S. FDA approved 46 new drugs, including 34 small molecule drugs and 12 biomolecule drugs, the highest total in 20 years, which is also a strong rebound after the low number of 22 in 2016.
The U.S. approved new drugs were dominated by antineoplastic and rare disease drugs, with 18 new drugs receiving orphan drug status, accounting for 39% of approved new drugs. The EU approved 19 new drugs for rare disease therapeutics, accounting for 38% of approved new drugs, either for small numbers of patients (as defined in the EU as diseases that fall into the 5 in 10,000 category) or for serious life- and health-threatening diseases, where drugs will receive priority review if the U.S. determines that they have the potential to make a substantial contribution to healthcare. In terms of therapeutic areas, antineoplastic drugs accounted for the majority in 2017, accounting for approximately 26%. In addition, anti-infective drugs also gained significant progress, including four antibacterial drugs and three antiviral drugs. Glaucoma, hepatitis C, psoriasis, diabetes and other therapeutic areas also gained breakthroughs.
Twenty of the approved new drugs in 2017 were identified for priority review, accounting for 43.5% of the approved new drugs. The U.S. also applies a variety of regulatory approaches to accelerate new drug development and approval, including fast track and breakthrough therapy designations. The accelerated review and the large proportion of new drug approvals for rare diseases are also related to the bumper crop of new drugs launched last year. From the perspective of clinical safety and effectiveness, developed countries also have different voices on accelerated review and approval, such as the Journal of the American Medical Association (JAMA) published an expert review in 2016, which concluded that 83 anti-cancer drugs listed from 2009 to 2014 were basically unreliable. two-thirds of the 222 listed new drugs counted in the article published in 2017 had safety concerns, 71 of which had safety issues and 61 required black boxes to warn of their safety. New drug users (physicians and patients) must therefore place a high priority on safety, availability and accessibility.
The first to receive FDA approval in 2017 was Synergy Pharmaceuticals’ new drug Trulance for the treatment of chronic idiopathic constipation. Another four new drugs for central nervous system diseases, Ocrevus for multiple sclerosis, Radicava for acromegaly, Xadago for Parkinson’s disease control, and Austedo for Huntington’s disease improvement, were approved by the FDA. new drugs were among the first in the world. For example, on December 11, 2017, the FDA approved the marketing of Medimetriks Pharmaceuticals’ Ozenoxacin, a new antibiotic for the treatment of children with impetigo over two months of age.
New drug development hits record milestone
2017 was a record-breaking year for global new drug development, with three milestones to remember.
One, on May 23, 2017, the FDA approved Merck Sharp & Dohme Keytruda for the treatment of patients carrying highly unstable or mismatch repair-deficient solid tumors, meaning that Keytruda became the first cancer treatment based not on the location of tumor development but on tumor markers, which is epoch-making in cancer history.
Second, on August 30, 2017, the FDA approved the marketing of Novartis’ Kymriah for the treatment of refractory or relapsed acute lymphoid leukemia in adolescents under the age of 25. kymriah thus became the world’s first marketed CAR-T therapy. kymriah is a genetically modified autologous T-cell immunotherapy, a customized treatment generated using the patient’s own T cells, whose safety and efficacy have been demonstrated in clinical trials. However, Kymriah is only highly efficacious in terms of remission rates within three months of treatment, and the treatment still has potentially serious side effects that may produce warnings about the risk of cytokine release syndrome (CRS) and may even be life-threatening due to CRS and neurological events, other serious side effects include severe infections, hypotension, acute kidney injury, fever and hypoxia, and also destruction of normal antibody-producing B cells, and there is a risk of increased prolonged infection.
Third, on December 19, 2017, the FDA approved the marketing of Spark Therapeutics’ gene therapy Luxturna for the treatment of hereditary retinopathy.Luxturna corrects genes directly in the patient’s body and is a true gene therapy, marking the official dawn of the gene therapy era. From the evidence of the safety and efficacy of Luxturna, the ability of patients in the test group to avoid obstacles in dim light was significantly improved after a single injection of Luxturna. However, the most common adverse effects such as conjunctival congestion, cataracts, high intraocular pressure, and retinal tears should not be ignored.