2019 Pipeline: Specialty Drug Approvals on the Horizon

Several new specialty therapies were approved this past year by the FDA. Looking ahead, there are a plethora of promising new treatments for a range of specialty conditions in the 2019 drug pipeline.
Here’s a look at some of the therapies slated for FDA approval over the next year.  
  1. Tenapanor (Ardelyx) for irritable bowel syndrome for constipation (IBS-C)
 Tenapanor is a first-in-class NH3E inhibitor being developed for IBS-C and hyperphosphatemia. According to Ardelyx, the company submitted a New Drug Application for IBS-C indication in September. Both phase 3 trials, T3MPO-1 and T3MPO-2, demonstrated tenapanor had a durable effect on reducing constipation and abdominal pain in patients with IBS-C. The favorable safety profile of tenapanor was further supported by the completed T3MPO-3 study.1
  1. Caplacizumab (Ablynx) for thrombotic thrombocytopenic purpura (TTP)
Caplacizumab is a nanobody against von Willebrand factor with both orphan and fast track designations. If approved, it will be the first drug specifically approved for acquired TTP, which is currently treated with plasmapheresis and immunosupressants. In the phase 3 HERCULES study, treatment with caplacizumab in addition to standard-of-care significantly shortened time to platelet count response.

The drug was already approved by the European Commission in September.2
  1. Siponimod (Novartis) for multiple sclerosis (MS) 
Siponimod, a follow-on sphingosine 1-phosphate modulator, is being developed for the treatment of secondary progressive MS (SPMS). Data from the EXPAND trial showed siponimod significantly reduced the risk of 3-month confirmed disability progression versus placebo, and also meaningfully delayed the risk of 6-month disability progression.
Novartis announced filing acceptance for the New Drug Application from the FDA in October. Regulatory action for siponimod is anticipated in the United States in March of 2019.3
  1. Sacituzumab govitecan (Immunomedics) for breast cancer 
In July, the FDA accepted Immunomedics supplemental Biologics License application for filing and granted priority review for sacituzumab govitecan for patients with metastatic triple-negative breast cancer (mTNBC) who previously received at least 2 prior therapies for metastatic disease. If approved, sacituzumab govitecan would be the first and only antibody-drug conjugate approved for the treatment of mTNBC.
According to Immunomedics, a regulatory decision is expected by January 2019.4 
  1. Quizartinib (Daiichi Sankyo) for acute myeloid leukemia (AML)
The FDA accepted a New Drug Application and granted Priority Review for quizartinib in November for the treatment of adult patients with relapsed/refractory FLT3-ITD AML. In a phase 3 study, oral quizartinib prolonged overall survival compared with chemotherapy in patients with the disease. The FDA is expected to make a decision on approval by May 25, 2019, according to Daiichi Sankyo.5

Read more on current and future developments in the oncology pipeline. 

  1. Ardelyx Submits New Drug Application for US Marketing Authorization of Tenapanor for IBS-C to US Food and Drug Administration [news release]. Ardelyx’s website. http://ir.ardelyx.com/news-releases/news-release-details/ardelyx-submits-new-drug-application-us-marketing-authorization. Ardelyx’s website. Accessed December 21, 2018.
  2. Cablivi (caplacizumab) approved in Europe for adults with acquired thrombotic thrombocytopenic purpura (aTTP) [news release]. Sanofi’s website. http://hugin.info/152918/R/2213684/863478.pdf. Accessed December 21, 2018.
  3. Novartis announces FDA and EMA filing acceptance of siponimod, the first and only drug shown to meaningfully delay disability progression in typical SPMS patients [news release]. Novartis’ website.  https://www.novartis.com/news/media-releases/novartis-announces-fda-and-ema-filing-acceptance-siponimod-first-and-only-drug-shown-meaningfully-delay-disability-progression-typical-spms-patients. Accessed December 21, 2018.
  4. FDA accepts biologics license application for filing and grants priority review for sacituzimab govitecan for the treatment of metastatic triple-negative breast cancer [news release]. Immunomedics’ website. https://www.immunomedics.com/our-company/news-and-events/fda-accepts-biologics-license-application-for-filing-and-grants-priority-review-for-sacituzumab-govitecan-for-the-treatment-of-metastatic-triple-negative-breast-cancer/. Accessed December 21, 2018.
  5. FDA Grants Priority Review for Daiichi Sankyo’s New Drug Application for FLT3 Inhibitor Quizartinib for Treatment of Patients with Relapsed/Refractory FLT3-ITD AML [news release]. Daiichi Sankyo’s website. https://www.daiichisankyo.com/media_investors/media_relations/press_releases/detail/006930.html. Accessed December 21, 2018.

Stay up to date on the latest news in specialty pharmacy by getting Specialty Pharmacy Times in your mailbox or inbox for free!

Click here to sign up for free for the bi-monthly Specialty Pharmacy Times print journal delivered to your address.

Click here to sign up for our email newsletters delivered every Monday, Wednesday, and Friday, in addition to breaking news alerts.

Click here to follow us on Facebook. 

Click here to follow us on Twitter. 

Click here to join our LinkedIn group. 

Related Articles

Scientific advances could lead to earlier prevention and treatment of infection, helping to avoid irreversible or severe chronic diseases.
In addition to a natural variation in genetic makeup, approximately 1 in 1000 people inherit a damaged or mutated copy of the BRCA1 gene from a parent.
Individuals with HIV maintained similar rates of viral suppression after switching from a 3-drug regimen to the 2-drug regimen of dolutegravir plus lamivudine.
Company Profile >
Industry Guide >
Market News >
Peer Exchange >
Conferences >
Subscribe >
Specialty Times Resources
About Us
Contact Us
Terms & Conditions
MJH Associates >
Pharmacy Times
American Journal of Managed Care
MD Magazine
Targeted Oncology
Physicians' Education Resource
Pharmacy & Healthcare Communications, LLC
2 Clarke Drive
Suite 100
Cranbury, NJ 08512
P: 609-716-7777
F: 609-716-4747

Copyright Specialty Pharmacy Times 2006-2019
Pharmacy & Healthcare Communications, LLC. All Rights Reserved.