As 2025 begins, we would like to reflect on last year’s regulatory developments in the biologics and biosimilars space.
Here are some of the top regulatory developments from 2024.
1. FDA Approvals in 2024
There were 17 FDA approvals of Biologic License Applications (“BLAs”), including for Autolus Inc.’s AUCATZYL (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, and AMTAGVI (lifileucel), the first approved cellular therapy for the treatment of adult patients with unresectable or metastatic melanoma. Additionally, the FDA approved Orchard Therapeutics’s LENMELDY (atidarsagene autotemcel), the first gene therapy to treat children with metachromatic leukodystrophy, a fatal genetic disease.
To close out the year, the FDA also approved on an accelerated basis Jazz Pharmaceuticals’s ZIIHERA (zanidatamab-hrii), the first and only dual HER2-targeted bispecific antibody and chemotherapy-free treatment for patients living with biliary tract cancer; Merus N.V.’s BIZENGRI (zenocutuzumab-zbco), the first and only approved treatment for adults with certain types of pancreatic adenocarcinoma or non-small cell lung cancer; and AstraZeneca’s IMFINZI (durvalumab), the first and only immunotherapy regimen for patients with limited-stage small cell lung cancer.
There were also 18 FDA approvals of biosimilar products, including 6 ustekinumab biosimilars and 5 aflibercept biosimilars in 2024 alone.
Ustekinumab approvals: The FDA approved Celltrion’s STEQEYMA (ustekinumab-stba) in December, Biocon’s YESINTEK (ustekinumab-kfce) in November, Accord’s IMULDOSA (ustekinumab-srlf) in October, Fresenius Kabi’s OTULFI (ustekinumab-aauz) in September, Samsung Bioepis’s PYZCHIVA (ustekinumab-ttwe) in July, and Alvotech’s SELARSDI (ustekinumab-aekn) in April. Additionally, in October 2024, the FDA approved SELARSDI for a new presentation—a single-dose vial for intravenous infusion—after previously approving it for use as a single-dose prefilled syringe for subcutaneous injections.
Aflibercept approvals: The FDA approved Amgen’s PAVBLU (aflibercept-ayyh) and Sandoz’s ENZEEVU (aflibercept-abzv) in August, Formycon’s AHZANTIVE (aflibercept-mrbb) in July, and Mylan/Biocon’s YESAFILI (aflibercept-jbvf) and Samsung Bioepis’s OPUVIZ (aflibercept-yszy) in May. After Amgen prevailed over Regeneron’s preliminary injunction motion—that has so far kept other aflibercept biosimilars off the market—and the Federal Circuit denied Regeneron’s motion for an injunction pending appeal, Amgen launched PAVBLU at the end of October 2024.
The FDA also approved the first biosimilar to Alexion’s SOLIRIS (eculizumab) on May 28, 2024. Amgen’s BKEMV (eculizumab-aeeb) was approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PHN) or atypical hemolytic uremic syndrome (aHUS).
2. Outcome of Year One of the Inflation Reduction Act’s Medicare Drug Price Negotiation Program
As we reported in 2023, the Inflation Reduction Act’s Medicare Drug Price Negotiation Program required that the Secretary of Health and Human Services (“HHS”) publish a list of 10 drugs that would be subject to negotiations with the government to determine the “maximum fair price” for the drug under Medicare. Among the 10 drugs that would be included in the first year of the program, HHS selected 3 biologics (ENBREL, STELARA, and FIASP and NOVOLOG products) with the remaining 7 being small-molecule drugs. On August 15, 2024, the Centers for Medicare & Medicaid Services (“CMS”) announced the negotiated “maximum fair prices” for the 10 drugs. The reductions in price ranged from 38 to 79 percent of the 2023 listed prices for the drugs. These new prices will take effect starting January 1, 2026.
3. FTC Issues Interim Report on Ongoing Study of Pharmacy Benefit Managers
On July 9, 2024, the Federal Trade Commission (“FTC”) released an interim report describing its ongoing study of pharmacy benefit managers (“PBMs”) and their impact on access to and affordability of medicines. In the report, the FTC discussed the role of PBMs as “middlemen, negotiating the terms and conditions for access to prescription drugs for hundreds of millions of Americans.” The FTC reported that the PBM industry is horizontally consolidated and vertically integrated, with the top six PBMs processing more than 90% of all prescriptions dispensed by U.S. pharmacies in 2023, and with the PBMs operating in a system from the drug private labeler all the way down to the pharmacy and health insurer. While recognizing that the interim report focused on “the impact of [the] changing market dynamics on the operation and vitality of the nation’s pharmacies,” the FTC explained that it also shared “initial evidence about PBM and brand pharmaceutical rebating practices that urgently warrant further scrutiny and potential regulation.” After the release of the interim report, Express Scripts, Inc., one of the largest PBMs in the United States, sued the FTC claiming that the report is defamatory, biased, and based on unfounded accusations. That litigation is ongoing.
4. FDA Guidances
Postapproval Manufacturing Changes to Biosimilar Products
On July 23, 2024, the FDA issued a draft guidance entitled “Postapproval Manufacturing Changes to Biosimilar and Interchangeable Biosimilar Products Questions and Answers.” The draft guidance is designed to “provide[] answers to commonly asked questions from applicants and other interested parties . . . regarding postapproval manufacturing changes . . . made to licensed biosimilars and licensed interchangeable biosimilars.”
Demonstrating Biosimilar Interchangeability With a Reference Product: Update
On June 20, 2024, the FDA announced a draft guidance which allows companies seeking a BLA for a biosimilar to be reviewed for interchangeability status to submit either switching study data or an assessment “of why the comparative analytical and clinical data provided in the application or supplement support a showing that the switching standard set forth in section 351(k)(4)(B) of the PHS Act has been met.” The draft guidance describes considerations regarding a switching study or studies intended to support a demonstration that a biological product is interchangeable with a reference product.
Promotional Communications of Prescription Biologics and Biosimilars
On April 25, 2024, the FDA published a notice of availability in the Federal Register for its revised draft guidance, entitled “Promotional Labeling and Advertising Considerations for Prescription Biological Reference Products, Biosimilar Products, and Interchangeable Biosimilar Products: Questions and Answers.” The updated guidance includes information on the general requirements for the content of FDA-regulated promotional communications about reference or biosimilar products and provides some examples to illustrate the FDA’s current recommendations for addressing reference, biosimilar, and interchangeable biosimilar products in product promotion.
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Looking ahead to the new year, the Big Molecule Watch team will continue to track and report on the key updates to the regulatory landscape. Among those updates, we will be watching for the potential approval of several proposed denosumab biosimilar products. We will also continue to monitor the CMS’s drug negotiation program, the FTC’s study of PBMs, and other regulatory activities arising from the new administration. For updates on these and more, please stay tuned.
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