Breakthrough Therapy Designations Set Quarterly Record
Executive Summary
US FDA granted designations for several products based on late-stage data; with at least 19 breakthrough therapy designations, five-year-old program sets another record in first quarter of 2018.
Breakthrough therapy designations continue to set records at the US FDA – this time another quarterly one.
Publicly announced receipts of the expedited pathway totaled 19 in the first quarter of 2018, according to data from Biomedtracker and the Pink Sheet's Performance Tracker, which tops the previous record of 18 in the second quarter of 2017. (See chart at end of story.)
Breakthrough therapy designations were created in 2013 and continue to be granted in record numbers, taxing FDA resources but allowing the agency to demonstrate its commitment and flexibility when it comes to reviews of innovative products. The 19designations from the first quarter are a continuance of the high totals from the second, third and fourth quarters of 2017. With three strong quarters of breakthrough therapy awards last year, 2017 became a record-setting year in terms of designations granted. (Also see "US FDA Sets Records With Breakthrough Therapy, Priority Review Designations In 2017" - Pink Sheet, 7 Jan, 2018.) Last year also featured a monthly record in May. (Also see "US FDA's Record-Breaking Month Of Breakthrough Therapy Designations" - Pink Sheet, 1 Jun, 2017.)
Now, the question is whether this increased pace will be maintained. Designations depend on industry research progress, as well as whether FDA has the resources to boost the pace any further.
A string of three recent breakthrough therapy designation announcements has been especially notable, as Bristol-Myers Squibb Co., Fennec Pharmaceuticals Inc. and Seattle Genetics Inc./Astellas Pharma Inc. received the expedited pathway after the pivotal trials for their respective treatments Opdivo (nivolumab)/Yervoy (ipilimumab), Pedmark (sodium thiosulfate) and enfortumab vedotin were well underway. The companies will see the benefits concentrated in the submission and review stages given the maturation of the development. (Also see "Breakthrough Status Arrives Late In Development Of Yervoy+Opdivo, Enfortumab Vedotin And Pedmark" - Pink Sheet, 30 Mar, 2018.)
Of the 19 total designations granted in the first quarter, the pivotal trial was at least underway, if not completed, for at least seven treatments: the Opdivo/Yervoy combination, Pedmark, enfortumab vedotin, Pfizer Inc.'s PF-04965842, Zogenix Inc.'s ZX008, Merck & Co. Inc.'s Lenvima (lenvatinib)/Keytruda (pembrolizumab) combination and Global Blood Therapeutics Inc.'s voxelotor. Additionally, the same was likely true for Novartis AG's Promacta (eltrombopag).
Pedmark is the only case where the company confirmed that the pivotal trial was also the basis for the breakthrough designation, but the same could be true for the Opdivo/Yervoy combination.
For the rest of the designations, it does not appear any of them were based on data earlier than Phase II.
Another notable breakthrough therapy status this year went to Alnylam Pharmaceuticals Inc.'s primary hyperoxaluria type 1 (PH1) candidate lumasiran. There is no FDA-approved treatment for PH1, an ultra-rare disease that involves excessive oxalate production, and lumasiran is the first PH1 investigational treatment to pick up a breakthrough therapy designation. (Also see "Keeping Track: A Burst Of Breakthroughs, A Priority Review For Keytruda, And Some Supplemental Submissions" - Pink Sheet, 18 Mar, 2018.)
Additionally, ZX008, designed for the adjunctive treatment of Dravet syndrome, appears to be the first with breakthrough status for the rare and severe form of epilepsy. There not yet an FDA-approved treatment for the disease, but the agency is already reviewing GW Pharmaceuticals PLC's cannibidiol product Epidiolex to treat seizures associated with Dravet syndrome and Lennox-Gastaut syndrome. (Also see "Keeping Track: DMD, RMAT, HIV, BTDs, Tc-99m – FTW" - Pink Sheet, 11 Feb, 2018.)
[Editor's note: The Breakthrough Therapy Designations chart on Pink Sheet's FDA Performance Tracker keeps track of BTDs with a sortable database that includes information on product candidates, the data supporting the BTD, and current clinical status.]
Priority Reviews Stay (Relatively) Hot
FDA additionally granted at least 20 priority reviews in the first quarter of 2018. Although the number is lower than that of the previous two quarters, it is still higher than most other quarterly totals in recent years.
Amicus Therapeutics Inc.'s Fabry disease candidate migalastat HCL was among the priority review recipients this year. FDA initially called for more data on gastrointestinal symptoms, which are common in Fabry disease patients, but surprisingly backtracked and allowed the company to file for accelerated approval. (Also see "Keeping Track: Vitaros Reenters The Wilderness, Remoxy Gives It A Fourth Try, And Two Novel Approvals" - Pink Sheet, 19 Feb, 2018.)
In four of the five previous years, priority review announcements have spiked in the third quarter. (Also see "Priority Review Designations In US: Third Quarter Spike Continues In 2017" - Pink Sheet, 6 Nov, 2017.)