The FDA, which approved 22 novel drugs last year, has given its approval to 34 drugs so far in 2017 including three in September. Key approvals this year include Novartis’s Kymriah (the first gene therapy in the United States), 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 and Sanofi’s eczema treatment, Dupixent, Tesaro’s PARP inhibitor, Zejula, and BioMarin’s Brineura (treatment of a specific form of Batten disease) among others. Quite a few 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 October.
Will Flexion Gain Approval for its First Drug?
Specialty pharma company, Flexion Therapeutics FLXN is awaiting FDA approval of its first product, Zilretta. The company, which is focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis (“OA”), expects a decision from the agency on Oct 6, 2017. Flexion is looking to get Zilretta approved for the treatment of OA related knee pain. Zilretta, a non-opioid, could provide OA patients with a new and alternative treatment option on approval. Flexion already has a sales leadership team in place and said that the pre-approval inspection of the Patheon manufacturing facility was concluded without any observations.
Flexion stock has gained 27.1% year to date, substantially outperforming the 3.6% rally of the industry it belongs to.
Alexion’s Soliris Label to be Expanded Again?
Alexion Pharmaceuticals ALXN is working on expanding the label of its flagship product, Soliris (eculizumab). Soliris, currently approved in the United States for serious ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (“PNH”) and atypical hemolytic uremic syndrome (aHUS), brought in sales of $1.6 billion in the first half of the year. Alexion has a key catalyst coming up with a decision from the FDA expected by Oct 23, 2017 regarding the approvability of Soliris for refractory generalized myasthenia gravis (gMG). EU approval for this indication came in August making it the first and only complement-based therapy approved for an ultra-rare subset of gMG. The gMG indication represents incremental growth opportunity for Soliris.
Alexion’s shares are up 14.7% year to date, compared to the industry’s 16.2% rally. Alexion is a Zacks Rank #3 (Hold) stock – you can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
CRL Likely for PTC’s Translarna
PTC Therapeutics PTCT is expecting a decision from the FDA regarding the approval status of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). While approved in the EU, Translarna is yet to gain approval in the United States. The company had initially filed a new drug application (“NDA”) in the United States in December 2015. However, in February 2016, the FDA issued a refusal to file (“RTF”) letter saying that the NDA was not sufficiently complete to allow a substantive review. The company was told that both the phase IIb and phase III ACT DMD studies were negative and did not provide sufficient evidence of effectiveness and adequate information about the abuse potential of Translarna. PTC filed a NDA in the first quarter of 2017 and was assigned an FDA action date of Oct 24, 2017.
However, chances of gaining approval seem low considering Translarna failed to gain the support of an FDA advisory panel last week. While the panel found the drug to be effective, they found the submitted data inconclusive and said that more data would be needed to support efficacy. Given this situation, we believe the FDA will issue a complete response letter (“CRL”) and ask for additional data.
PTC’s shares are up 83.4% year to date, compared to the industry’s 3.6% rally.
Apart from the above three companies, quite a few other companies are awaiting decisions from the FDA. Industry bellwether, Johnson & Johnson JNJ is seeking FDA approval for a 10mg dose of Xarelto for reducing the risk of recurrent venous thromboembolism (VTE). With the FDA granting priority review, a response should be out on Oct 28, 2017.
Cancer-focused company, TESARO TSRO is expecting a decision from the FDA for its intravenous (“IV”) formulation of rolapitant on Oct 25, 2017. The company had received a CRL earlier this year for the IV formulation.
Specialty pharma company, AcelRx Pharmaceuticals ACRX expects a response from the FDA regarding its NDA for Dsuvia (sufentanil sublingual tablet, 30 mcg) on Oct 12, 2017. Dsuvia is under review for the treatment of moderate-to-severe acute pain in medically supervised settings.
Meanwhile, Aerie Pharmaceuticals’s AERI Rhopressa will be evaluated by an FDA advisory panel for the treatment of patients with open-angle glaucoma or ocular hypertension on Oct 13, 2017.
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