UniQure (NASDAQ: QURE) announced on January 30th that their lead asset, AMT-060 received Breakthrough Therapy designation (BTD) by the FDA. AMT-060 is a gene therapy being evaluated in patients with Hemophilia B. With this designation come several commercialization advantages resulting in expedited development and review times. To qualify for BTD, the investigational therapy must treat a life threatening disease and have preliminary clinical evidence demonstrating a substantial improvement over existing therapies.
In 2016, the FDA received 106 requests for BTD: 46 were granted, 48 were denied, and 12 were withdrawn.
· Sponsors can interact more frequently with experienced FDA personnel and gain input on trial design
· Sponsors can propose alternative trial designs that are more efficient (shorter duration, lower enrollment, use of historical control, single arm trials)
· The FDA may assign a project lead to help facilitate clinical review, leading to shorter review times
· Designation enables rolling review, and can allow for priority review, and accelerated approval of application
Based on the criteria set forth by the FDA, it seems reasonable that UniQure will be able to enroll a single arm trial that would qualify for priority review and accelerated approval.
UniQure is one of AMP Biotech Research's quarterly picks for Q1 2017. AMP is a biotech research firm that publishes a quarterly list of companies with attractive valuations and near-term catalysts. To see all of AMP's picks for Q1 2017, go to ampbioresearch.com.
Find the original press release here: http://www.uniqure.com/investors-newsroom/press-releases.php
For more on Breakthrough Therapies, see here: http://www.fda.gov/RegulatoryInformation/Legislation/SignificantAmendmentstotheFDCAct/FDASIA/ucm329491.htm
Disclosure: I am/we are long QURE.