FDA Watchlist: Catalysts To Look Out For This February

2017 turned out to be a notable year for the pharma and biotech industry with the FDA giving its nod to 46 novel drugs as well as several biological license applications ("BLA") including for path-breaking cancer treatments like Kymriah and Yescarta. This number compares favorably not only to 2016 when just 22 novel drugs were approved, but to earlier years as well.

Other key approvals last year include Lilly's Verzenio (advanced or metastatic breast cancer), Gilead's Vosevi (hepatitis C virus), Puma's Nerlynx (to reduce the risk of breast cancer returning), J&J's Tremfya (moderate-to-severe plaque psoriasis), Regeneron/Sanofi's Kevzara (rheumatoid arthritis), Roche's multiple sclerosis treatment, Ocrevus, Regeneron REGN and Sanofi's eczema treatment, Dupixent, Tesaro's PARP inhibitor, Zejula, and BioMarin's BMRN Brineura (treatment of a specific form of Batten disease) among others. Many of these drugs have blockbuster potential.

With the drug development process being lengthy and time-consuming and requiring the utilization of a lot of funds and resources, key pipeline events including data readouts and regulatory updates are of paramount importance and could act as major catalysts. Here is a look at a few important regulatory events scheduled for the month of February.

FDA Decision for AMAG's Makena and Feraheme: AMAG Pharmaceuticals AMAG is seeking FDA approval for a subcutaneous auto-injector version of Makena - the drug-device combination product, if approved on the PDUFA date of February 14, would offer the convenience of using a ready-to-administer subcutaneous auto-injector while providing patients with an alternative option to an intramuscular injection. Makena is currently available as an intramuscular injection for reducing the risk of preterm birth in women who are pregnant with one baby and who spontaneously delivered one preterm baby in the past.  

The approval of this product would help AMAG extend the patent life of Makena which is scheduled to lose orphan drug exclusivity on February 3. While AMAG is ready to launch an authorized generic along with a partner in February itself, the company does not expect a generic competitor to enter the market until later in 2018. If the drug-device combination product is approved, the company will apply for Orange Book listing of the patents for the device.

Meanwhile, the FDA is expected to issue a decision regarding the label expansion of Feraheme on February 2. AMAG is looking to expand the label beyond the current chronic kidney disease ("CKD") indication to include all eligible adult patients with iron deficiency anemia ("IDA").

Priority Review for Gilead's HIV Treatment: Gilead Sciences GILD is also awaiting a decision from the FDA this month for its new drug application ("NDA") for an investigational, fixed-dose combination of bictegravir (50 mg) ("BIC"), an integrase strand transfer inhibitor, and emtricitabine/tenofovir alafenamide (200/25 mg) (FTC/TAF), a dual-NRTI backbone, for the treatment of HIV-1 infection. The NDA is under priority review with a decision expected on February 12. With the company's hepatitis C virus ("HCV") franchise seeing declining sales, Gilead is focusing on its HIV franchise for growth. The company's HIV portfolio includes drugs like Genvoya, Truvada, Atripla, Descovy, Odefsey, Complera and Stribild which contributed approximately $9.5 billion to total sales in the first nine months of 2017. Gilead's TAF-based products like Genvoya, Descovy and Odefsey remain key growth drivers.

Will Vertex Boost its CF Portfolio with an FDA Approval This Month? Vertex Pharmaceuticals VRTX is a key player in the cystic fibrosis ("CF") market with two products, Orkambi and Kalydeco, in its CF portfolio. The company is looking to strengthen its position in this area further with the potential approval of a tezacaftor/ivacaftor combination treatment that is under priority review with a decision from the FDA expected on February 28. The combination treatment is under review for use in CF patients (12 years and older) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation that is responsive to the treatment.

The approval of this combination would be a major boost for Vertex's CF portfolio which is expected to deliver revenues in the range of $2.1 - $2.15 billion in 2017. Currently, about 31,000 patients are eligible for treatment under the company's CF portfolio with the number expected to grow to 44,000 in 2018 on the back of EU reimbursement for Orkambi, label expansion for the drug and the potential approval of the tezacaftor/ivacaftor combination.

Vertex's shares are up 94% over the last one year, outperforming the 10% rally of the industry it belongs to. Vertex is a Zacks Rank #3 (Hold) stock - you can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Valeant Pharmaceuticals International, Inc. VRX also expects to hear from the FDA about the approval status of Plenvu, a bowel cleanser used in preparation for colonoscopies (FDA action date: February 13). Moreover, companies like KemPharm and Apricus expect a response from the FDA this month for their resubmitted NDAs. While KemPharm has a PDUFA date of February 23 for Apadaz (an immediate-release, abuse-deterrent hydrocodone/acetaminophen combination product candidate), Apricus will get to know the approval status of its resubmitted Vitaros (topically delivered treatment for erectile dysfunction) NDA on February 17.

Meanwhile, the FDA's Anesthetic and Analgesic Drug Products Advisory Committee will be meeting on February 14 to discuss Pacira Pharmaceuticals's PCRX supplemental new drug application (sNDA) for Exparel (bupivacaine liposomal injectable suspension), to produce local analgesia and as a nerve block to produce regional analgesia.

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