Introduction
Over the years, the Prescription Drug User Fee Act (PDUFA) has authorized the USFDA to collect user fees from industry to facilitate the review process for certain human drug applications. The fifth authorization of PDUFA (2012-2017) focused mainly on advancing public safety and upgrading the benefit/risk assessment of new medicines. PDUFA VI was passed for fiscal years 2018 to 2022. The PDUFA VI ensures that FDA will continue to receive a source of stable and consistent funding during fiscal years 2018-2022 that will allow the agency to fulfill its mission to protect and promote public health by helping to bring to market critical new medicines for patients.
This review discusses the highlights of NDA approvals in 2017. Figure 1 provides a month-wise comparison of NDA approvals in the year 2016 and 2017. Except in January, February, May and July, all other months in 2017 received more NDA approvals. Overall, 185 NDAs were approved in 2017 compared to 131 approvals in 2016.
Figure 1. A month-wise comparison of NDA approvals in 2016 and 2017.A dosage form is one of the important parameters for every product. In 2017, injections were the majority (37%) over all other dosage forms followed by tablets (27%) (Figure 2). Injections include, lyophilized powder for injection, a solution, powder for solution, etc. Tablets contribute to most of the solid dosage forms. Liquids include oral solutions, suspensions, ophthalmic solutions, otic solutions, etc.
Figure 2. Number of percent dosage form reported in the year 2017Figure 3 depicts a list of companies receiving five or more NDA approvals in 2017. Genentech topped the list with 13 NDA approvals in 2017. This number is much higher compared to the number of approvals in 2016 (6 approvals). Merck and AstraZeneca received 11 and 9 NDA approvals, respectively. Janssen was at the top in 2016 with seven NDA approvals but received only five NDA approvals in 2017.
Figure 3. Companies with five or more NDA approvals in 2017FDA’s Center for Drug Evaluation and Research’s (CDER’s) used several regulatory pathways to expedite the development and approval of novel drugs in 2017. These pathways utilize a range of approaches that can enhance development efficiency and shorten timelines. Figure 4 shows some key measures approved by FDA. A total of 30 NDAs were approved for new indications while 28 approvals were approved as the first FDA approved therapy. Breakthrough approvals are drugs with preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least one clinically significant endpoint (e.g., study result) over other available therapies for serious or life-threatening diseases for which there is unmet medical need. The Accelerated Approval program allows FDA more flexibility in deciding which endpoints can be used to approve a drug that offers a benefit over current treatments for a serious or life-threatening illness.
Figure 4. Regulatory pathways to expedite the development and approval of novel drugs in 2017In 2017, FDA’s new drug therapy approvals helped a wide range of patients suffering from many different medical conditions - from rare disorders to common diseases - to gain new hope for improved quality of life, and in some cases, improved chances of surviving life-threatening illnesses. FDA approved a total of 38 products for various cancer therapies including acute lymphoblastic leukemia, Merkel cell carcinoma, certain forms of relapsed or refractory acute myeloid leukemia, certain forms of lymphoma, recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, and specific forms of liver, breast, and colorectal cancer. Infectious diseases and neurological disorder received 12 and 11 approvals, respectively (Figure 5). Infectious disease includes serious skin infections, complicated urinary tract infections including kidney infections, chronic hepatitis C, cytomegalovirus infection and the first therapy in the United States to treat Chagas disease - a rare parasitic disease. Neurological disorders includes tardive dyskinesia, Duchenne muscular dystrophy, multiple sclerosis, amyotrophic lateral sclerosis, schizophrenia, and Parkinson’s disease. Rare diseases include sickle cell disease, hemophilia A with inhibitors, giant cell arteritis and Batten disease.
Figure 5. Top in class categoriesNew and Notable Formulations
Table 1 includes notable new formulations as well as other new formulations of a drug for which the product’s active ingredient is already FDA-approved. Three of the new formulations have properties that are intended to deter abuse of these highly addictive medications.
Table 1. New and Notable formulations approved in 2017
Biosimilars Approved in 2017
FDA-approved biosimilar products are highly similar to the brand and have no clinically meaningful differences in terms of safety, purity and potency (safety and effectiveness) from an already FDA-approved biological product, called the reference product. Biological products are highly complex, and often used to treat patients with serious and life-threatening conditions. The law allowing FDA to approve biosimilars was designed to create competition, increase consumer choice, and support greater access to important therapies.
Table 2. Biosimilar NDA approvals in 2017
Special Approvals
Brineura™ from BioMarin Pharmaceutical: Brineura™ (cerliponase alfa) was approved as a treatment for a specific form of Batten disease - a rare disease that can cause progressive neurological impairments, including seizures, visual problems/blindness, personality and behavior changes, dementia, and loss of the ability to walk, talk, and communicate.
Hemlibra® from Genentech: Hemlibra® (emicizumab) was approved for the prevention of bleeding or to reduce the frequency of bleeding episodes in patients with hemophilia A who have developed antibodies called Factor VIII inhibitors. This is the first non-blood product approved for this condition.