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5 FDA approval decisions to watch in the 1st quarter

Credit: Yujin Kim / MedTech Dive, original photo courtesy of U.S. Food and Drug Administration

The Food and Drug Administration cleared for market 48 new drugs through its main review office last year. Though that's lower than the 59 approvals seen in 2018, the agency's decisions still provided more treatment options for patients living with cystic fibrosis, sickle cell disease and rare muscular disorders.

Notably, the agency ended 2019 with a flurry of earlier-than-expected decisions, bolstering the 2018 count with several drugs it was scheduled to finish review on this year.

It's unclear how or if the approval stream will change in the new year and decade. The first quarter of 2020, though, may prove a bellwether in the near term. Between January and March, the FDA is slated to make calls on a handful of impactful drugs, including these five.

1. Aimmune Therapeutics' Palforzia for peanut allergy

The odds are good that, before January closes out, Aimmune's Palforzia will receive approval to treat one of the most common food allergies. That's because the committee responsible for advising the FDA on whether or not to approve new allergy medicines recently voted in favor of the drug's effectiveness and safety. While the agency doesn't have to follow the guidance of advisory committees, it typically does.

Getting to market isn't the last hurdle for Aimmune, however. The company needs to secure insurance coverage, a task analysts expect will be more difficult if the drug comes at a high list price. Consensus on Wall Street seems to be that the company will set a list price somewhere in the range of $5,000 to $10,000 for the first six months of therapy, with lower costs thereafter.

Aimmune's case could also be complicated by clinical results that showed patients on Palforzia needed epinephrine injections about twice as frequently as those on placebo. The drug's labeling and risk mitigation strategy will therefore be closely watched.

"Overall, we remain cautious on the peanut category as a whole and believe out year consensus sales estimates for Palforzia seem overly optimistic given the complexity of the therapy and tolerability profile for a preventative treatment," Stifel analyst Derek Archila wrote in an early November investor note. The investment bank expects Palforzia sales to hit $65 million in 2020 and $800 million in 2025.

2. Esperion Therapeutics' bempedoic acid for high cholesterol

Lowering so-called bad cholesterol has been a profitable endeavor for some pharmaceutical companies. At its peak, Pfizer's Lipitor, now generic, was bringing in revenue of nearly $13 billion a year.

Lipitor is part of a drug class called statins, which continue to hold a significant portion of the cholesterol drug market despite the entry of newer medicines. Amgen's Repatha and Sanofi and Regeneron's Praluent, each a member of another drug class called PCSK9 inhibitors, have continued to fall short of sales expectations because of their relatively high cost.

Yet with roughly a third of the U.S. population having high levels of bad cholesterol, and statins being insufficient for some patients, companies remain interested in carving out market share for statin alternatives or additions. Amgen, Sanofi and Regeneron have shaved down the list price on their medicines, while Novartis in November agreed to shell out almost $10 billion to acquire The Medicines Company and its experimental PCSK9 drug.

Esperion may provide an additional option with bempedoic acid, a prodrug that inhibits an enzyme involved in cholesterol production. The Michigan-based company should find out by Feb. 21 whether regulators have cleared its drug. Another approval application for the combination of bempedoic acid and ezetimibe — the active ingredient in Merck & Co.'s Zetia — has a review deadline of Feb. 26.

Though Esperion's drug raised some safety concerns, a series of five late-stage trials support its efficacy. The FDA also didn't require an advisory committee meeting for the drug, which analysts said bodes well for its chances of approval.

3. Blueprint Medicine's avapritinib for cancer

Blueprint may get its first marketable drug with avapritinib, a treatment for patients who have certain kinds of gastrointestinal stromal tumors. The FDA is scheduled to make an approval decision by Feb. 14.

If approved, Blueprint’s drug would join a growing wave of targeted cancer therapies to reach market. That field has experienced an uptick in investment as well as big pharma interest, as evidenced by the recent buyouts of Loxo Oncology, Array BioPharma and Ignyta.

Avapritinib, however, has been the cause of some investor worries over the last few months. In late October, Blueprint disclosed that the FDA had split the drug’s approval application — which was going after two different portions of the adult gastrointestinal cancer population — into two parts. The decision keeps one approval decision on track for the Feb. 14 deadline, but pushes the other back three months.

Analysts at SVB Leerink noted how this delay is problematic for Blueprint, as it could narrow the amount of time avapritinib has on market before the potential entry of a rival medicine from Deciphera Pharmaceuticals.

4. Biohaven Pharmaceutical's rimegepant for migraine

Allergan achieved an industry first last month, when its drug Ubrelvy — part of a drug class known as CGRP inhibitors — gained approval as an oral acute treatment for episodic migraine. Until that point, the FDA had only approved CGRP inhibitors like Amgen's Aimovig and Eli Lilly's Emgality, both injections, to prevent these severe headaches from happening.

But Ubrelvy may not have much time without direct competition. Biohaven's rimegepant, which, like Ubrelvy, is an oral CGRP inhibitor, could also gain approval in episodic migraine treatment before the end of the first quarter.

Piper Jaffray analyst Tyler Van Buren wrote in a recent note that, in light of Ubrelvy's approval, regulators are likely to sign off on Biohaven's drug as well. The investment bank models $40 million in rimegepant ​sales next year and more than $1 billion by 2024.

5. Bristol-Myers Squibb's ozanimod for multiple sclerosis

Bristol-Myers may have closed on its historic $74 billion Celgene acquisition, but its payments aren't yet complete. Former Celgene shareholders may still take home an additional $9 per share if three of the biotech's experimental drugs secure approval over the next two years.

One of those drugs is ozanimod, which targets the same receptor protein as Novartis' blockbuster multiple sclerosis medicine Gilenya.

Ozanimod has already faced one setback, when the FDA initially refused to review its application because of incomplete non-clinical pharmacology sections. Celgene resubmitted the application a year later, in March 2018, and expects to see an approval decision by March 25.

Despite the longer-than-expected regulatory timeline, ozanimod remains supported by positive clinical readouts. Salim Syed of Mizuho Securities USA contends that, "based on the data in the public domain, we don't see a reason for the drug to not get approved."

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