Key FDA Actions Anticipated in 2018

The FDA approved a number of new therapies and indications for a variety of conditions this past year (read our 2017 recap here). With 2017 behind us, we’re looking ahead at the next year for what’s on the horizon in pharmacy.

These are some of the upcoming drugs slated for review by the FDA in 2018.

1. Biologics

Several Biologics License Applications (BLAs) and supplemental Biologics License Applications (sBLA) have action dates set by the FDA for 2018.

Erenumab (Aimovig, Amgen and Novartis)¹

The FDA has accepted for review the BLA for erenumab for the prevention of migraine in patients experiencing 4 or more migraine days per month. If approved, Aimovig is expected to be the first and only monoclonal antibody targeting the calcitonin gene-related peptide receptor, specifically designed for the prevention of migraine.

In phase 2 and phase 3 trials, erenumab demonstrated a reduction in the number of migraine-affected days, disability, and acute medication use for patients with episodic and chronic migraine.

Action date: May 17, 2018

Plasminogen (Ryplazim, Prometic Life Sciences)²

The FDA accepted Prometic Life Sciences’ BLA for its plasminogen replacement therapy in October 2017, having granted a priority review status.

Previous data reported from a phase 2/3 trial demonstrated that Ryplazim treatment consistently replaced and maintained the plasminogen concentration at an appropriate level and that it resolved all lesions in patients treated.

Action date: April 14, 2018

Burosumab (Ultragenyx Pharmaceutical Inc.)³

The FDA accepted the BLA for burosumab for the treatment of pediatric and adult patients with X-linked Hypophosphatemia (XLH) in October 2017 and has granted priority review status. Burosumab also received breakthrough therapy designation from the agency for the treatment of XLH in pediatric patients 1 year of age and older.

Burosumab is being developed by Ultragenyx, Kyowa Hakko Kirin, and Kyowa Kirin International.

Action date: April 17, 2018

Denosumab (Prolia, Amgen)⁴

The FDA accepted the sBLA for denosumab in October 2017 for the treatment of patients with glucocorticoid-induced osteoporosis based on a phase 3 study evaluating denosumab compared with risedronate in patients receiving glucocorticoid treatment.

Action date: May 28, 2018.

2. New Drugs

Halobetasol propionate and tazarotene (IDP-118, Valeant)⁵

The FDA accepted the new drug application (NDA) submission for IDP-118 (halobetasol propionate and tazarotene) lotion, an investigational topical treatment for plaque psoriasis, from Ortho Dermatologics (a division of Valeant Pharmaceuticals International).

If approved, it will be the first and only topical lotion that contains a unique combination of halobetasol propionate and tazarotene in 1 formulation for the treatment of plaque psoriasis in adult patients, allowing for potentially expanded duration of use.

Action date: June 18, 2018

Aripiprazole Lauroxil NanoCrystal Dispersion (Alkermes)⁶

Aripiprazole Lauroxil NanoCrystal Dispersion is a novel, investigational product designed for initiation onto aripiprazole lauroxil (Aristada) extended-release injectable suspension for the treatment of schizophrenia. The FDA accepted Alkermes' NDA in November 2017.

If approved, administration in conjunction with a single oral dose of 30 mg aripiprazole will replace the need for 3 weeks of concomitant oral aripiprazole with the first dose of Aristada. It is designed for initiation onto any dose or duration of Aristada.

Action date: June 30, 2018

Fostamatinib disodium (Tavalisse, Rigel Pharmaceuticals)⁷

Rigel Pharmaceuticals filed its NDA for the use of fostamatinib disodium (Tavalisse), an oral investigational drug designed to inhibit SYK kinase, in patients with chronic or persistent immune thrombocytopenia (ITP).

According to Rigel, fostamatinib may address an underlying autoimmune cause of ITP by impeding platelet destruction, unlike other therapies that modulate the immune system in different ways to stimulate platelet production.

Action date: April 17, 2018

Buprenorphine sublingual spray (INSYS Therapeutics)⁸

INSYS Therapeutics is seeking approval for its novel formulation of buprenorphine as a sublingual spray for the management of moderate-to-severe acute pain. Its NDA was based on a pivotal trial that met its primary efficacy endpoint and several pharmacokinetic studies.

According to INSYS, potential benefits of buprenorphine include a ceiling effect for respiratory depression and, compared to other opioids frequently used in this indication, less abuse potential, less cognitive impairment, and less constipation.

Action date: July 28, 2018

TX-004HR (TherapeuticsMD)⁹

TherapeuticsMD is seeking FDA approval for TX-004HR, its investigational, applicator-free estradiol vaginal softgel capsule for the treatment of moderate to severe vaginal pain during sexual intercourse, a symptom of vuvular and vaginal atrophy due to menopause.

Action date: May 29, 2018

3. Expanded Indications

Ferumoxytol (Feraheme, AMAG Pharmaceuticals)¹⁰

AMAG is seeking to broaden the existing label for ferumoxytol to include the treatment of all adults with iron deficiency anemia (IDA) who have an intolerance or unsatisfactory response to oral iron. The current indication is limited to the treatment of IDA in adults with chronic kidney disease.

Action date: February 2, 2018

Ciprofloxacin otic suspension (OTIPRIO, Otonomy Inc.)¹¹

Ciprofloxacin otic suspension is currently approved by the FDA for use during tympanostomy tube placement surgery in pediatric patients, but Otonomy Inc. is seeking approval for an expanded indication to include its use for the treatment of acute otitis externa in patients with tympanostomy tubes.

Action date: March 2, 2018

Bupivacaine liposome injectable suspension (Exparel, Pacira Pharmaceuticals)¹²

The FDA accepted Pacira’s resubmission of its sNDA seeking expansion of the Exparel label to include administration via nerve block for prolonged regional analgesia. Exparel is currently indicated for single-dose infiltration into the surgical site to produce postsurgical analgesia.

Action date: April 6, 2018

References

1. Aimovig™ (erenumab) Phase 3 STRIVE Data Published In The New England Journal Of Medicine Demonstrate Significant, Sustained Efficacy In Migraine Prevention [news release]. Amgen's website. http://www.amgen.com/media/news-releases/2017/11/aimovig-erenumab-phase-3-strive-data-published-in-the-new-england-journal-of-medicine-demonstrate-significant-sustained-efficacy-in-migraine-prevention/. Accessed December 29, 2017.

2. Prometic Announces FDA Acceptance of its Biologics License Application for Plasminogen (RYPLAZIM™) [news release]. Prometic's website. http://www.prometic.com/prometic-announces-fda-acceptance-of-its-biologics-license-application-for-plasminogen-ryplazim/. Accessed December 27, 2017.

3. Ultragenyx and Kyowa Hakko Kirin Announce FDA Acceptance and Priority Review Designation of Burosumab's Biologics License Application [news release]. Ultragenyx's website. http://ir.ultragenyx.com/releasedetail.cfm?ReleaseID=1043322. Accessed December 27, 2017.

4. FDA Accepts Supplemental Biologics License Application For Prolia® (Denosumab) In Glucocorticoid-Induced Osteoporosis [news release]. Amgen's website. http://www.amgen.com/media/news-releases/2017/10/fda-accepts-supplemental-biologics-license-application-for-prolia-denosumab-in-glucocorticoidinduced-osteoporosis/. Accessed December 27, 2017.

5. Novel Investigational Topical Lotion with Unique Formulation Targets the Treatment of Plaque Psoriasis [news release]. Valeant's website. http://ir.valeant.com/news-releases/2017/09-05-2017-141939465. Accessed December 27, 2017.

6. Alkermes’ New Drug Application for Investigational Product Designed for Initiation Onto ARISTADA® Accepted for Filing by U.S. FDA [news release]. Alkermes' website. http://phx.corporate-ir.net/phoenix.zhtml?c=92211&p=irol-corporateNewsArticle&ID=2317379. Accessed December 27, 2017.

7. FDA Accepts Rigel's New Drug Application for TAVALISSE™ (fostamatinib disodium) for the Treatment of Chronic ITP [news release]. Rigel's website. http://ir.rigel.com/phoenix.zhtml?c=120936&p=irol-newsArticle&ID=2281550. Accessed December 27, 2017.

8. FDA Accepts New Drug Application (NDA) for Buprenorphine Sublingual Spray from INSYS Therapeutics [news release]. INSYS's website. http://investors.insysrx.com/phoenix.zhtml?c=115949&p=irol-newsArticle&ID=2321406. Accessed December 27, 2017.

9. TherapeuticsMD Announces FDA Acceptance of New Drug Application and Prescription Drug User Fee Act (PDUFA) Date for TX-004HR [news release]. TherapeuticsMD's website. https://ir.therapeuticsmd.com/news-releases/news-release-details/therapeuticsmd-announces-fda-acceptance-new-drug-application-and. Accessed December 27, 2017.

10. AMAG Announces U.S. FDA Filing Acceptance and Six-Month Review of Feraheme® (ferumoxytol) for the Treatment of All Adult Patients With Iron Deficiency Anemia [news release]. AMAG's website. https://www.amagpharma.com/news/amag-announces-u-s-fda-filing-acceptance-and-six-month-review-of-feraheme-ferumoxytol-for-the-treatment-of-all-adult-patients-with-iron-deficiency-anemia/. Accessed December 27, 2017.

11. Otonomy Announces FDA Acceptance of OTIPRIO® Supplemental New Drug Application Filing for Acute Otitis Externa [news release]. Otonomy's website. http://investors.otonomy.com/phoenix.zhtml?c=234082&p=irol-newsArticle_Print&ID=2286970. Accessed December 27, 2017.

12. Pacira Pharmaceuticals Announces FDA Acceptance of sNDA for EXPAREL as a Nerve Block to Produce Regional Analgesia [news release]. Pacira's website. http://investor.pacira.com/phoenix.zhtml?c=220759&p=irol-newsArticle&ID=2309425. Accessed December 27, 2017.