With the numerous advancements being made in medicine every day, it’s easy to forget how long it can take for a treatment to go from the lab to the hands of a patient. Some drugs are researched for years before they are presented to the FDA for approval, which can take up to a year itself. Many drugs, however, are on the cusp of landing on the market. Here are the current top 10 new drugs that have made it to the final step in the process and are up for FDA approval.
- Ixekizumab for Psoriasis
- CVT-301 for Parkinson’s
- VC-01 for Type 1 Diabetes
- SPN-810 for ADHD
- AG-221 for Patients With Acute Myelogenous Leukemia That Harbor an IDH2 Mutation
- OS2966 for Glioblastoma
- Rayaldee for Secondary Hyperparathyroidism
- Mocetinostat for Large B-Cell Lymphoma
- GlaxoSmithKline’s Ebola Vaccine
- Ruxolitinib Daily for Hair Loss in Patients With Alopecia
Eli Lilly’s ixekizumab is one of many new experimental psoriasis treatments battling for center stage and is currently in the lead. Ixekizumab surpassed Pfizer and Amgen’s blockbuster Enbrel in a phase 3 study, with 31% to 41% of patients attaining clear skin after 12 weeks of treatment compared with just 5% to 7% of Enbrel patients. Lilly reported on August 28 that it would seek marketing approval for the drug in the first half of 2015, with analysts forecasting annual sales of $600 million by 2020. The treatment works by blocking an inflammation-causing protein called IL-17. Other key competitors developing similar treatments include Novartis, Celgene, Amgen, AstraZeneca, Merck, and Johnson & Johnson.
Civitas Therapeutics, a privately held, Massachusetts-based pharmaceutical company developing and commercializing transformative therapeutics, announced positive results from a phase 2b clinical trial of a treatment for Parkinson’s disease patients called CVT-301. The treatment is a self-administered inhaled formulation of levodopa (L-dopa) that provides rapid relief from intermittent debilitating motor fluctuations associated with the disease, known as “off” episodes. Off episodes affect over half of all patients on an oral L-dopa treatment regimen because its efficacy is compromised by delayed absorption and excessive variability in the circulating plasma drug concentrations inherent to the oral delivery route. CVT-301 incorporates L-dopa by delivering a precise dose to the deep lung for rapid and predictable absorption. Going forward, Civitas Therapeutics will be undertaking a $55 million venture aimed at getting the treatment through to phase 3 and on to the market.
ViaCyte, Inc., a privately held regenerative medicine company, announced that the FDA has accepted its Investigational New Drug (IND) Application for its cell replacement therapy called VC-01, which was created to treat type 1 diabetes. The treatment consists of pancreatic progenitor cells, called PEC-01, that are derived from a proprietary human embryonic stem cell line. According to a press release, the PEC-01 cells are implanted under the skin and are “designed to mature and further differentiate into insulin-producing beta and other endocrine cells that regulate blood glucose in a manner similar or identical to the islets that normally comprise the endocrine pancreas.” ViaCyte has plans to move forward with a phase 1/2 clinical trial that will evaluate VC-01 directly in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function.
The FDA has granted fast-track designation for Supernus Pharmaceuticals, Inc.’s SPN-810 for the treatment of impulsive aggression in attention deficit hyperactivity disorder (ADHD). Supernus Pharmaceuticals is a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system diseases. The company reported positive topline results in a phase 2b trial of SPN-810 and is expected to enter phase 3 testing, with patient dosing commencing during 2015. Currently, there are no products approved for treating impulsive aggression in ADHD patients, making SPN-810 an important game changer. The fast-track designation allows for the early submission of some sections of the marketing application, with the potential for an expedited review category for the New Drug Application (NDA).
The FDA also granted fast-track designation to Agios Pharmaceuticals’ AG-221 for the treatment of patients with acute myelogenous leukemia (AML) who harbor an isocitrate dehydrogenase 2 (IDH2) mutation. Agios Pharmaceuticals is a leader in the fields of cancer metabolism and rare genetic metabolism disorders. The treatment is an oral, selective, potent IDH2 mutant inhibitor currently in a phase 1 clinical trial in patients with advanced hematologic malignancies. “We are committed to working with our partner Celgene Corporation to get this medicine to patients as soon as possible,” said Agios chief medical officer Dr. Chris Bowden.
OS2966, a neutralizing anti-CD29 monoclonal antibody to treat glioblastoma produced by biopharmaceutical firm OncoSynergy, was recently granted orphan drug designation by the FDA’s Office of Orphan Products Development. The treatment inhibits multiple mechanisms of resistance promoted by interactions with the tumor microenvironment. Preclinical data suggest that the drug may be active against numerous solid cancers, including glioblastoma. Glioblastoma is the most common and deadliest primary adult brain tumor.
OPKO Health, Inc. recently announced successful top-line results from phase 3 trials of Rayaldee, a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease and vitamin D insufficiency. The trial involved 213 patients who were randomized to receive 6 months of treatment with either Rayaldee or a placebo. All patients had vitamin D insufficiency, which was corrected in 96% of those who were treated with Rayaldee. “Rayaldee is designed to fill a void in the treatment armamentarium of nephrologists and endocrinologists who care for pre-dialysis chronic kidney disease patients,” said Dr. Charles W. Bishop, CEO of OPKO’s Renal Division. “Controlled trials have shown that over-the-counter and prescription vitamin D supplements are ineffective in treating SHPT in these patients and are, at best, unreliable in correcting vitamin D insufficiency.” An NDA submission is expected for the drug by the end of 2014.
Another drug on the FDA’s list for orphan drug designation is Mirati Therapeutics, Inc.’s mocetinostat, a spectrum-selective HDAC inhibitor for patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer with specific genetic mutations in histone acetyltransferases (HATs). The drug reverses aberrant acetylation resulting from HAT mutations and is predicted to halt tumor progression and reduce tumor burden in patients. Mocetinostat is currently in phase 2 clinical trials in combination with Vidaza as a treatment for intermediate- and high-risk myelodysplastic syndrome. Phase 2 studies of the drug as a single agent in patients with mutations in HATs in bladder cancer and DLBCL are also underway. Data from the trials are expected to be available by the end of 2014.
One of the more urgent drugs that is being rushed through trials is GlaxoSmithKline’s Ebola vaccine. As of August 28, 2014, Ebola has infected at least 3069, killing 1552 people in West Africa. This outbreak is the largest in history. Several experimental vaccines for the virus have been previously tested, but none have made it to the finish line. The next promising vaccine to go to trial is being developed by the National Institutes of Health and GlaxoSmithKline and is going to be tested in healthy people to determine if it is safe and induces an adequate immune response starting September 1.
Columbia University Medical Center researchers announced that ruxolitinib, an approved treatment in the US normally used for myelofibrosis, has completely reversed baldness caused by alopecia areata in 3 patients. Alopecia areata is a type of hair loss that occurs when the immune system mistakenly attacks hair follicles. The treatment would not work on the more common male-pattern baldness, which is linked to hormones. After completely reversing hair loss in mice, ruxolitinib has moved on to testing in 3 human patients with alopecia areata. All 3 showed dramatic hair regrowth after treatment for up to 5 months. “Additional clinical trials are needed to test the effectiveness of this drug in more patients in larger studies,” said study author Angela M. Christiano, a professor of dermatology and genetics at Columbia. “However, for patients with alopecia areata, this is an exciting result, because it offers a potential new class of drugs that have not been tried before in this disease, with some promising early results.”
- Blaszczak-Boxe A. Drug could regrow hair in some with hair loss. Fox News website. August 18 2014. http://www.foxnews.com/health/2014/08/18/drug-could-regrow-hair-in-some-with-hair-loss.
- Civitas Therapeutics announces positive phase 2b results for CVT-301, inhaled levodopa for the treatment of Parkinson’s disease [press release]. Civitas Therapeutics website. April 28, 2014. http://civitastherapeutics.com/cms/sites/default/files/news/Civitas%20Phase%202b%20 Results%20Release%20FINAL%2014%200428.pdf.
- Civitas Therapeutics initiates phase 2a clinical study of CVT-301, an inhaled L-dopa for Parkinson’s disease. BusinessWire website. June 15, 2012. http://www.businesswire.com/news/ home/20120615005092/en/Civitas-Therapeutics-Initiates-Phase-2a-Clinical-Study#.U_5H5vldV8E.
- Mocetinostat overview. Mirati Therapeutics website. http://www.mirati.com/lead-oncology-assets/mocetinostat.
- NIH to launch human safety study of Ebola vaccine candidate. National Institute of Allergy and Infectious Diseases website. August 28, 2014. http://www.niaid.nih.gov/news/newsreleases/ 2014/Pages/EbolaVaxCandidate.aspx.
- OncoSynergy’s OS2966 glioblastoma treatment gets FDA orphan drug status. PBR website. August 13, 2014. http://regulatoryaffairs.pharmaceutical-business-review.com/news/oncosynergys-os2966-glioblastoma-treatment-gets-fda-orphan-drug-status-130814-4343157.
- Pierson R. Update 2-Lilly psoriasis drug impresses; battle nears with Amgen, Novartis. Reuters website. August 21, 2014. http://in.reuters.com/article/2014/08/21/elililly-study-idINL2N0QR16V20140821.
- Rayaldee phase 3 trial meets primary endpoints. OPKO website. August 11, 2014. http://investor.opko.com/releasedetail.cfm?ReleaseID=865608.
- Supernus receives FDA fast track designation for SPN-810. Market Watch website. August 14, 2014. http://www.marketwatch.com/story/supernus-receives-fda-fast-track-designation-for-spn-810-2014-08-14.
- ViaCyte, Inc. announces FDA acceptance of IND to commence clinical trial of VC-01™ candidate cell replacement therapy for type 1 diabetes. PR Newswire website. August 19, 2014. http://www.prnewswire.com/news-releases/viacyte-inc-announces-fda-acceptance-of-ind-to-commence-clinical-trial-of-vc-01-candidate-cell-replacement-therapy-for-type-1-diabetes-271829971.html.