5 FDA decisions to watch in the first quarter

09 Jan 2023
www.biopharmadive.com
Drug ApprovalClinical ResultPhase 3AcquisitionAccelerated Approval
The downturn that struck the biotechnology industry in 2022 wasn’t just about tumbling stock prices. The sector also won only 37 new drug approvals from the Food and Drug Administration’s main review office last year, its lowest total since 2016.
Yet the number of drugs the FDA clears each year can fluctuate, and industry observers see reasons for optimism. The 2022 approvals of Amylyx Pharmaceuticals’ ALS drug and Provention Bio’s Type 1 diabetes treatment show an “increasing permissiveness” for drugs with mixed or limited data, RBC Capital Markets analysts wrote in December. Drug rejections and FDA-mandated trial stoppages were down compared to 2021. The regulator also cleared three gene therapies for inherited diseases, a sign that Commissioner Robert Califf “seems open to innovation,” they wrote.
That sentiment could be put to the test early in 2023. The FDA is weighing decisions on another drug for Alzheimer’s as well as a gene therapy for hemophilia A.
Here are five decisions to watch in the first quarter:
Eisai and Biogen’s Alzheimer’s drug Leqembi, which U.S. regulators conditionally cleared last week, could soon have company. Following closely behind it is donanemab, a similarly acting drug from Eli Lilly that could reach market sometime within the next two months.
An accelerated approval for donanemab would give Lilly something to show for the many years it’s invested in Alzheimer’s research. But the company may face an uphill battle winning market share, at least in the near-term.
Leqembi has already been shown to slow disease progression in a large Phase 3 trial, albeit modestly. Donanemab’s application, by comparison, is largely based on results from smaller mid-stage studies. Late-stage data are expected toward the middle of the year, potentially months after the drug is available.
In the meantime, doctors could have tough decisions to make about which drug to prescribe — and Lilly may have to tread carefully in choosing a price. Biogen ran into fierce resistance from insurers when it priced its earlier Alzheimer’s drug, Aduhelm, at $56,000 a year despite mixed results in clinical testing. Eisai chose a lower price of $26,500 a year for Leqembi, setting a new bar.
Until the FDA grants a full clearance, however, neither Leqembi nor donanemab, if approved, are likely to see much use under restrictive coverage rules put in place by Medicare. — Jonathan Gardner
Before being acquired last year by two private equity firms, biotech company Radius Health was primarily known for an osteoporosis drug called Tymlos that it struggled to sell. Its second act, a breast cancer medicine called elacestrant, may have a better chance at commercial success.
Elacestrant is one of a group of medicines, known as SERDs for short, that block and degrade hormone receptor proteins and are administered as pills. The hope is these drugs could replace fulvestrant, an injectable medicine that’s widely prescribed for breast cancer patients whose disease is driven by hormones. Yet their prospects are unclear. Only elacestrant has succeeded in a pivotal trial. Two similar drugs from Roche and Sanofi failed.
An approval by the FDA’s Feb. 17 deadline could give the Italian pharmaceutical company Menarini Group, which licensed elacestrant from Radius in 2020, the first crack at a potentially substantial market. Menarini is seeking approval of the drug for patients with an advanced, common form of breast cancer known as ER-positive, HER2-negative. Elacestrant would be the first branded endocrine therapy for this patient group in more than two decades.
Analysts at Stifel, citing discussions with breast cancer experts in a December research note, said adoption in the second-line setting could be strong if the drug is approved. But that could depend on the price Menarini chooses, and whether the FDA approves it for all patients, or only the subset who appear to benefit the most, they wrote.
Menarini may not have the market to itself for long, either. AstraZeneca, Lilly and Arvinas each have rival medicines in advanced testing. — Ben Fidler
2023 could be the year BioMarin finally brings Roctavian, its gene therapy for hemophilia A, to market in the U.S. But BioMarin may still have one more obstacle to overcome on its already lengthy journey to an FDA approval.
BioMarin has spent years developing Roctavian for hemophilia A, the most common form of the chronic blood disease and a condition caused by a lack of a clotting protein. Like Hemgenix, the hemophilia B gene therapy the FDA approved in November, Roctavian is meant to be a long-lasting treatment that boosts levels of clotting protein, protecting patients from spontaneous bleeds and ridding them of the need to take other drugs. It’s shown the ability to do so in clinical testing, but also signs that its benefits may wane with time.
BioMarin’s data has been enough to sway European regulators, which approved Roctavian last August. Yet the company has had a tougher time convincing the FDA. The agency rejected Roctavian in 2020, asking for more data on how the treatment’s effects held up. BioMarin subsequently collected those data and filed a new application. But the company recently signaled even that review may extend beyond its March 31 deadline as the FDA analyzes full three-year results from Roctavian’s main trial, which were released on Sunday.
Despite the lingering uncertainty, analysts are generally optimistic. The approval of Hemgenix shows the FDA “appear[s] to be more comfortable with hemophilia gene therapies,” SVB Securities analysts wrote in December. RBC analysts also noted in December that the agency had reversed an earlier decision to hold an advisory committee meeting, removing a potential hurdle.
An approval would keep BioMarin ahead of Pfizer and Roche, which are also developing hemophilia A gene therapies. — Delilah Alvarado
Twice now, the FDA has turned back attempts by Acadia to secure an expanded approval for a Parkinson’s disease drug it sells as Nuplazid. In April 2021 and again last August, the agency rejected the company’s applications to extend use of Nuplazid to treat psychosis related to dementia.
Acadia’s regulatory difficulties have weighed on the company, as shares have sunk by nearly two-thirds since the initial signs trouble with the first application.
But the company is hoping for a different outcome with its drug trofinetide, which it has been developing for a neurodevelopmental disorder called Rett syndrome. Its application is now under review by the FDA, with a target decision date of March 12.
In clinical testing, treatment with trofinetide improved scores on two assessments of Rett syndrome symptoms compared to placebo among girls and young women with the disorder.
If approved, trofinetide would become the first treatment for Rett, giving patients a needed option. For Acadia, success would also be a boon, potentially offsetting struggles the company has had selling Nuplazid in its approved Parkinson’s disease indication. An approval could also come with a special regulatory voucher that can be resold.
Acadia acquired North American rights to trofinetide in a 2018 deal with Neuren Pharmaceuticals. If the drug is approved and the FDA issues a voucher, Neuren would be due one-third of its value. — Ned Pagliarulo
Gilead is best known for its HIV and hepatitis C medicines, two highly lucrative businesses that were catalyzed by acquisitions.
The California biotech has hopes for similar success with its $21 billion purchase of Immunomedics two-plus years ago, the largest acquisition in the company’s history. The deal brought in a cancer drug called Trodelvy that Gilead views as the linchpin in its push into oncology, a field of research the company historically hasn’t taken part in.
But, so far, analysts aren’t convinced the deal was worth its high price. Trodelvy is currently approved for a tough-to-treat “triple-negative” form of breast cancer. Broadening its use into the most common form of breast cancer, known as HR-positive and HER2-negative, could help boost its prospects.
Initial results from an important clinical trial were positive, but viewed as showing only a modest benefit. Follow-up data revealed treatment led to three months longer survival.
The FDA is currently reviewing Gilead’s request to expand Todelvy’s approval to cover use in this setting, for adults with previously treated locally advanced or metastatic breast cancer that can’t be removed surgically. A decision is due in February.
Even if an approval is granted, Gilead faces tough competition from another breast cancer drug called Enhertu. Developed by AstraZeneca and Daiichi Sankyo, Enhertu has shown strong results in testing and was recently approved by the FDA for use in treating cancers that are considered to be “HER2 low.”
Analysts currently expect Trodelvy would be used after Enhertu in this kind of breast cancer. — Ned Pagliarulo
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