2019 Pipeline: Specialty Drug Approvals on the Horizon


Looking ahead, there are a plethora of promising new treatments for a range of specialty conditions in the 2019 drug pipeline.

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.

  • 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

  • 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

  • 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

  • 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

  • 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.


  • 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.
  • 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.
  • 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.
  • 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.
  • 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.

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