Priority Reviews At US FDA: From Third Quarter Spikes To A Third Quarter Trough
Executive Summary
Priority review announcements saw significant dip in third quarter 2018, while they usually experience a spike during the period; breakthrough therapy designations remained in double digits.
Priority review designation announcements regarding applications pending at the US FDA have generally spiked in the third quarter over the last several years, with 2016 being the exception. But not only have they failed to peak in third quarter of 2018; it looks like the three-month stretch will prove to be a notable trough.
After achieving respective totals of at least 21 and 28 in the first and second quarters of this year, sponsors announced only 12 priority review designations in the third quarter of 2018, according to Biomedtracker data, which marks a decrease of roughly 57%. (See chart below.)
Priority review announcements similarly bottomed out in the third quarter of 2016 before shooting back up in the fourth quarter. The drop from the two previous quarters of that year, however, was far less than it was for this year's third quarter.
The large drop could be the result of a slew of different factors, including market dynamics and the progress of industry research.
But previous trends suggest this year's fourth quarter will experience a rebound to some extent, as has generally been the case with previous quarters following a trough. To match the 2017 record of 83 priority review announcements, there would need to be 22 such announcements in the fourth quarter of 2018. (See chart below). (Also see "US FDA Sets Records With Breakthrough Therapy, Priority Review Designations In 2017" - Pink Sheet, 7 Jan, 2018.) Given the high totals of recent quarters, such a number is not unattainable.
There are several applications in FDA's pipeline not yet accepted for review that will likely receive priority status, including:
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Intra-Cellular Therapies Inc.' lumateperone for the treatment of schizophrenia;
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Janssen Pharmaceutical Cos.'s erdafitinib for the treatment of patients with locally advanced or metastatic urothelial cancer (UC) and certain FGFR genetic alterations whose tumors have progressed after prior chemotherapy;
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Janssen's esketamine for treatment-resistant depression in adults; and
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Karyopharm Therapeutics Inc.'s Selinexor for the treatment of patients with penta-refractory multiple myeloma.
Many sponsors, however, do not announce the filing of an application until disclosing it has been accepted for review.
Breakthrough Designations Remain High
Since the second quarter of 2017, breakthrough therapy designation announcements have remained in the double digits as the program has grown in popularity. (Also see "Breakthrough Therapy Designations Set Quarterly Record" - Pink Sheet, 3 Apr, 2018.)
Sponsors made 13 such announcements in the third quarter of 2018, bringing the year's sum to 43. That number is just 12 shy of the high-water mark set in 2017 of 55. (Also see "US FDA Sets Records With Breakthrough Therapy, Priority Review Designations In 2017" - Pink Sheet, 7 Jan, 2018.)
If the recent quarters are any indication, it appears 2018 still has a legitimate chance leapfrog that total.
Pfizer Inc. is the most recent to announce the receipt of breakthrough therapy status, picking up the designation for its 20-valent pneumococcal conjugate (20vPnC) vaccine candidate PF-06482077 designed to prevent invasive disease and pneumonia caused by Streptococcus pneunomiae serotypes. (Also see "Keeping Track: An Approval For Ajovy, A CRL For Ruconest, And Some Submissions from J&J" - Pink Sheet, 23 Sep, 2018.)
What has made 2018 noteworthy is the amount of breakthrough therapy designations being announced late in the development process. (Also see "Breakthrough Status Arrives Late In Development Of Yervoy+Opdivo, Enfortumab Vedotin And Pedmark" - Pink Sheet, 30 Mar, 2018.)
The third quarter was no different, with another string of companies disclosing the expedited pathway when a pivotal trial was already underway including Genentech Inc.'s Tecentriq (atezolizumab)/Avastin (bevacizumab) combination, Eisai Co. Ltd. and Merck & Co. Inc.'s Lenvima (lenvatinib)/Keytruda (pembrolizumab) combination, Daiichi Sankyo Co. Ltd.'s quizartinib, Array Biopharma Inc.'s combination of Braftovi (encorafenib), Mektovi (binimetinib) and Erbitux (cetuximab), Y-mAbs Therapeutics Inc.'s naxitamab/GM-CSF combination and AstraZeneca PLC and Amgen Inc.'s tezepelumab.
Another notable breakthrough announcement during the third quarter came from Genentech for it's anti-IgE antibody Xolair (omalizumab) for the prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies. So far, breakthrough designations for food allergy medications have been limited to those designed specifically for peanut allergies. (Also see "Keeping Track: A Long-Awaited Approval For Generic EpiPen, Cancer Product Labels Expand And Constrict" - Pink Sheet, 19 Aug, 2018.)
So long as the breakthrough therapy designation count says high, the priority review count likely will as well, as breakthrough status is a strong indicator of priority status. However, the breakthrough totals are dependent on factors such as scientific advancement, and whether FDA has the resources to continue growing the pace.
(See the Breakthrough Therapy chart on the FDA Performance Tracker for information on products receiving the designation.)