Burosumab – 2018 Drug Pipeline Review

X-Linked Hypophosphatemia (XLH) is a rare genetic musculoskeletal disorder that is characterized by low levels of phosphate and has the potential to lead to bone abnormalities or rickets.

Current conventional treatment of XLH includes daily phosphate and active vitamin D doses to serve as replacement therapy, but this does not treat or correct the underlying disease. Therefore, drug candidates under development have become increasingly necessary to target the true mechanisms of this debilitating disease.

Burosumab (KRN23) is a fully human monoclonal antibody that is being developed by Kyowa Hakko Kirin Co., Ltd in collaboration with Ultragenyx Pharmaceutical Inc. for the treatment of X-Linked Hypophosphatemia in both pediatrics and adults. XLH is precipitated by excess levels of fibroblast growth factor 23 (FGF23), a factor that works to regulate phosphate homeostasis, and is known to “waste” phosphate in XLH. Burosumab, administered as a subcutaneous injection, targets and restores the normal functioning of FGF23.

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Burosumab (Kyowa Hakko Kirin Co., Ltd.)

Regulatory

 

Status

Pre-Registration

PDUFA

4/17/2018

  Orphan Drug Designation
 

X-Linked Hypophosphatemic Rickets

12/14/2009

 

Osteomalacia

6/15/2017

  Breakthrough Therapy Designation

6/28/2016

  FDA Priority Review

10/10/2017

  Patent Expiration

2/5/2029

   
Indications X-Linked Hypophosphatemic Rickets

Submitted for Approval (BLA)

   
Efficacy X-Linked Hypophosphatemic Rickets

Efficacy reported in Phase III Trials

Forecasted Sales

(in millions)2

2018

$55

References:

  1. Clinical Trial data obtained at https://clinicaltrials.gov/ct2/home Accessed: March 5, 2018.
  2. GlobalData Pharma Intelligence Center https://pharma.globaldata.com Accessed: February 26, 2018.
  3. Kyowa Kirin International PLC. “Kyowa Kirin and Ultragenyx Announce Crysvita® (Burosumab) Receives Conditional Marketing Authorisation in Europe for the Treatment of X-Linked Hypophosphatemia in Children.” PR Newswire, 23 Feb. 2018, www.prnewswire.com/news-releases/kyowa-kirin-and-ultragenyx-announce-crysvita-burosumab-receives-conditional-marketing-authorisation-in-europe-for-the-treatment-of-x-linked-hypophosphatemia-in-children-674945013.html.
  4. XLH Network. http://xlhnetwork.org/what-is-xlh/faq/ Accessed March 9, 2018.

 


For an overview of FDA PDUFA dates as of March 2018 click here

Coauthored by: Kristen Ciampi, PharmD Candidate

Kristen is a sixth-year pharmacy student at the University of Rhode Island. Kristen is completing her studies through an advanced pharmacy practice rotation at RJ Health Systems prior to graduating with a PharmD degree in May of 2018.

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