Drug Approvals

New Law Could Make Accelerated Approval Stricter, If FDA Enforces It

The FDA’s accelerated approval pathway will see major changes following December’s passage of the Consolidated Appropriations Act, 2023 (2023 CAA), the latest version of the annual legislation that funds the federal government. Experts say that the changes should force drugmakers to be more diligent about proving the clinical value of their early-stage pharmaceuticals — as long as the FDA uses its enforcement powers.

Stakeholders across the health care system have criticized the FDA’s approach to accelerated approval in recent years. The agency’s critics say that it has taken a lax approach, granting accelerated approval to drugs of dubious clinical value, which ultimately costs the health care system billions. In particular, the FDA’s move to grant accelerated approval to Biogen Inc.’s Alzheimer’s drug Aduhelm (aducanumab) faced criticism from researchers, medical practitioners, health systems and insurers. In addition, a recent congressional report documented ethical lapses by agency employees during the accelerated approval process for Aduhelm, and the HHS Office of Inspector General is currently investigating the agency’s decision.

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News Briefs: Lilly Alzheimer’s Drug Fails to Win Accelerated Approval

The FDA declined to grant accelerated approval for Alzheimer’s disease treatment donanemab and requested additional data from its manufacturer, Eli Lilly & Co. said on Jan. 19. The move comes on the heels of the FDA’s decision to bestow accelerated approval on another Alzheimer’s treatment called Leqembi (lecanemab) from Biogen and Eisai — although a National Coverage Determination issued for Biogen’s Aduhelm (aducanumab) significantly limits who can access any drug in that class that receives accelerated approval. In its response letter to Lilly, the FDA cited “the limited number of patients with at least 12 months of drug exposure data provided in the submission” for accelerated approval as its justification for denying the application, and the agency asked Lilly to provide data from at least 100 patients who received a minimum of 12 months of continued treatment on donanemab. Lilly said it expects to unveil results from its Phase III confirmatory trial in the second quarter of this year, and that “will form the basis of donanemab's application for traditional approval shortly thereafter.”

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New Law Could Make Accelerated Approval Stricter, If FDA Enforces It

The FDA’s accelerated approval pathway will see major changes following December’s passage of the Consolidated Appropriations Act, 2023 (2023 CAA), the latest version of the annual legislation that funds the federal government. Experts say that the changes should force drugmakers to be more diligent about proving the clinical value of their early-stage pharmaceuticals — as long as the FDA uses its enforcement powers.

Stakeholders across the health care system have criticized the FDA’s approach to accelerated approval in recent years. The agency’s critics say that it has taken a lax approach, granting accelerated approval to drugs of dubious clinical value, which ultimately costs the health care system billions. In particular, the FDA’s move to grant accelerated approval to Biogen Inc.’s Alzheimer’s drug Aduhelm (aducanumab) faced criticism from researchers, medical practitioners, health systems and insurers. In addition, a recent congressional report documented ethical lapses by agency employees during the accelerated approval process for Aduhelm, and the HHS Office of Inspector General is currently investigating the agency’s decision.

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FDA Approves Gilead’s First-in-Class HIV Drug Sunlenca

The FDA recently approved a new HIV drug for a small patient population desperately in need of treatments. And the twice-yearly medication’s annual price came below the level that respondents to a Zitter Insights poll said they would consider a good value.

On Dec. 22, the FDA approved Gilead Sciences, Inc.’s Sunlenca (lenacapavir) for the treatment, in combination with other antiretroviral(s), of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection who are failing their current antiretroviral regimen due to resistance, intolerance or safety considerations. The agency gave the first-in-class capsid inhibitor priority review, fast track and breakthrough therapy designations.

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For Now, New Alzheimer’s Treatment Is Subject to Limited Medicare Coverage

Marking the second approval of an Alzheimer’s disease treatment aimed at slowing cognitive decline by reducing amyloid plaque buildup on the brain, the FDA on Jan. 6 granted accelerated approval to Biogen and Eisai, Co., Ltd.’s Leqembi (lecanemab-irmb). For now, the drug remains subject to the restrictive National Coverage Determination that Medicare initially gave its predecessor, Aduhelm (aducanumab-avwa), and similar Alzheimer’s treatments last year, but CMS could consider broader coverage if the drug receives traditional FDA approval.

The drug is indicated for the treatment of Alzheimer’s disease in people with mild cognitive impairment or mild dementia. Patients must have confirmed presence of amyloid beta pathology before starting treatment. The FDA gave the humanized immunoglobulin gamma 1 monoclonal antibody fast track, priority review and breakthrough therapy designations. Japanese pharmaceutical company Eisai developed the drug and will co-market it with its U.S. partner Biogen, which launched Aduhelm in 2021 and had to halve its price from $56,000 a year to $28,000. Eisai estimates the cost of Leqembi will be $26,500 per year, though the actual price could vary by patient.

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New FDA Approvals: FDA Gives Accelerated Approval to Mirati’s Krazati

Dec. 12: The FDA gave accelerated approval to Mirati Therapeutics, Inc.’s Krazati (adagrasib) for the treatment of adults with KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test (see below brief), who have received at least one systemic therapy. Dosing for the tablet is 600 mg twice daily. The drug is priced at $19,750 per month.

Dec. 12: The FDA approved two companion diagnostics for Krazati (see above brief): Agilent Technologies Inc.’s Agilent Resolution ctDx FIRST assay and Qiagen N.V.’s Qiagen therascreen KRAS RGQ PCR kit. A liquid biopsy next-generation sequencing assay, the Agilent test detects genomic alterations in circulating tumor DNA from plasma. The Qiagen test, first approved July 6, 2012, is a tissue-based assay.

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FDA Approved Four New Gene Therapies, Other Novel Agents in 2022

While the FDA may not have approved the most drugs in a year in 2022, it still gave the green light to a number of agents, many of them specialty medications, as well as granted additional indications to existing therapies. The FDA’s Center for Drug Evaluation and Research (CDER) approved 39 new molecular entities in 2022, and the Center for Biologics Evaluation and Research (CBER) approved 12 biologic license applications, including four new gene therapies in the second half of the year. AIS Health, a division of MMIT, asked some industry sources what the most notable 2022 FDA approvals were and why they were so important.

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FDA Approves Alzheimer’s Agent Leqembi Shortly After Release of Congressional Aduhelm Report

Less than two years after the FDA approved the first treatment for Alzheimer’s that was aimed at targeting the underlying disease process, it has approved a second similar agent. Payers are likely to cover the drug, one industry expert says, but they almost certainly will try to place restrictions on their coverage.

On Jan. 6, the FDA gave accelerated approval to Eisai Co., Ltd. and Biogen Inc.’s Leqembi (lecanemab-irmb) for the treatment of Alzheimer’s disease in people with mild cognitive impairment or mild dementia stage of disease. People must have confirmed presence of amyloid beta pathology before starting treatment. The agency gave the humanized immunoglobulin gamma 1 monoclonal antibody fast track, priority review and breakthrough therapy designations.

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For Alzheimer’s Drug Leqembi, Coverage Hinges on Full FDA Approval

Via the FDA’s accelerated approval pathway, Biogen and Eisai got the green light from federal regulators on Jan. 6 to take their Alzheimer’s drug Leqembi (lecanemab) to market. But sources tell AIS Health, a division of MMIT, that uptake and coverage of the medication will likely be underwhelming until it receives full FDA approval — a designation the manufacturers have already filed to acquire.

Leqembi represents the drugmakers’ second crack at offering a breakthrough Alzheimer’s drug, as they launched Aduhelm (aducanumab) in 2021 after it received its own accelerated approval nod despite considerable controversy over the medication’s safety and efficacy. Both drugs are monoclonal antibodies that aim to slow cognitive decline in Alzheimer’s patients by reducing amyloid plaque buildup on the brain of patients in early stages of the disease.

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2022 Saw Continued Approvals, Uptake of Biosimilars

Following the FDA’s March 6, 2015, approval of the first biosimilar in the United States — Zarxio (filgrastim-sndz) from Novartis Pharmaceuticals Corp.’s Sandoz unit — these agents have continued to gain market share, even though not all that have been approved have launched. And while the European Union approved its first biosimilar, Sandoz’s Omnitrope (somatropin), almost a decade earlier on April 12, 2006, the U.S. outpaced the EU in biosimilar approvals in the eight-year post-launch period 40 to 15. Last year continued the trend, with the FDA approving seven new biosimilars. AIS Health, a division of MMIT, asked some industry experts about the agents’ impact over the past year.

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