Vertex Pharma Gains FDA Breakthrough Therapy Designation For Kidney Disease Drug

Vertex Pharmaceuticals VRTX received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its drug candidate inaxaplin (VX-147) for the treatment of patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS).

The FDA grant was supported by the Phase 2 clinical study of inaxaplin in patients with APOL1-mediated FSGS, a form of AMKD.

Additionally, the European Medicines Agency (EMA) has granted inaxaplin Priority Medicines (PRIME) designation for APOL1-mediated chronic kidney disease (AMKD).

The PRIME designation was supported by the clinical proof-of-concept data readout from the Phase 2 clinical study of inaxaplin in APOL1-mediated FSGS.

FDA grants Breakthrough therapy designation (BTD) to expedite the development and review of drugs for serious or life-threatening conditions. The BTD requires preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

Vertex Pharmaceuticals shares are currently higher at $272.75 in the regular trading session.

Posted In: FDA GrantBiotechNewsHealth CareFDAGeneral

Ad Disclosure: The rate information is obtained by Bankrate from the listed institutions. Bankrate cannot guaranty the accuracy or availability of any rates shown above. Institutions may have different rates on their own websites than those posted on Bankrate.com. The listings that appear on this page are from companies from which this website receives compensation, which may impact how, where, and in what order products appear. This table does not include all companies or all available products.

All rates are subject to change without notice and may vary depending on location. These quotes are from banks, thrifts, and credit unions, some of whom have paid for a link to their own Web site where you can find additional information. Those with a paid link are our Advertisers. Those without a paid link are listings we obtain to improve the consumer shopping experience and are not Advertisers. To receive the Bankrate.com rate from an Advertiser, please identify yourself as a Bankrate customer. Bank and thrift deposits are insured by the Federal Deposit Insurance Corp. Credit union deposits are insured by the National Credit Union Administration.

Consumer Satisfaction: Bankrate attempts to verify the accuracy and availability of its Advertisers' terms through its quality assurance process and requires Advertisers to agree to our Terms and Conditions and to adhere to our Quality Control Program. If you believe that you have received an inaccurate quote or are otherwise not satisfied with the services provided to you by the institution you choose, please click here.

Rate collection and criteria: Click here for more information on rate collection and criteria.