Drug Approvals

Feb. 3: The FDA expanded the patient population for Takeda’s Takhzyro (lanadelumab-flyo) to include the prevention of hereditary angioedema attacks in people at least 2 years old. The agency initially approved the plasma kallikrein inhibitor on Aug. 3, 2018. Dosing of the subcutaneous injectable in people at least 12 years old is 300 mg every two weeks. In people at least 6 and less than 12, dosing is 150 mg every two weeks, and for people 2 years old and younger than 6, dosing is 150 mg every four weeks. Drugs.com lists the price of one single-dose 300 mg/2 mL subcutaneous solution as more than $26,165.

Feb. 8: The FDA granted another indication to Regeneron Pharmaceuticals, Inc.’s Eylea (aflibercept) for the treatment of retinopathy of prematurity in preterm infants. The agency first approved the drug on Nov. 18, 2011. Dosing of the vascular endothelial growth factor (VEGF) inhibitor for the newest indication is 0.4 mg via intravitreal injection; treatment may be given bilaterally on the same day. Injections may be repeated with an interval of at least 10 days. Drugs.com lists the price of a 40 mg/ml intravitreal solution as more than $1,957.

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. The medication comes with both potential advantages and disadvantages for patients, providers and payers, say industry experts.

On Dec. 22, the FDA approved Gilead Sciences, Inc.’s Sunlenca (lenacapavir) for the treatment, in combination with other antiretroviral(s) (ARVs), 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.

Covis Pharma Group said on March 7 that it will voluntarily stop selling Makena (hydroxyprogesterone caproate), a drug that aims to reduce preterm births and that has become a flashpoint in the debate over the FDA’s accelerated approval pathway. Makena was granted accelerated approval in 2011, but subsequent clinical trials failed to demonstrate its effectiveness, leading FDA advisers last fall to recommend withdrawing the drug from the market. “While we stand by Makena’s favorable benefit-risk profile, including its efficacy in women at highest risk of preterm birth, we are seeking to voluntarily withdraw the product and work with the FDA to effectuate an orderly wind-down,” Covis Chief Innovation Officer Raghav Chari, Ph.D., said in a statement. The move comes ahead of an anticipated final decision on Makena’s status by FDA Commissioner Robert Califf and Chief Scientist Namandjé Bumpus.

The FDA recently approved the first gene therapy for bladder cancer, Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg). Almost three-quarters of oncologists surveyed by Zitter Insights expressed at least moderate interest in the agent, and payers said they likely will manage the drug to label.

On Dec. 16, the FDA approved Adstiladrin for the treatment of adults with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. The agency gave the novel adenovirus vector-based gene therapy priority review, breakthrough therapy and fast track designations. Dosing is once every three months into the bladder via a urinary catheter. The company said it expects the therapy to be available in the second half of 2023.

The FDA on Feb. 2 granted approval for a self-administered version of Amgen Inc. and AstraZeneca plc’s Tezspire (tezepelumab-ekko), a biologic used as a preventive maintenance therapy for severe asthma. Pharmacy experts tell AIS Health, a division of MMIT, that the drug’s high price tag and high clinical thresholds mean that adoption is unlikely to spike in the short term.

The new version of Tezspire, a human monoclonal antibody, can be self-administered by patients through a pre-filled pen. Doses last four weeks. Tezspire is “the only biologic approved for severe asthma with no phenotype (e.g., eosinophilic or allergic) or biomarker limitation within its approved label,” per an Amgen press release. It was first approved in December 2021, But previously, the drug had to be administered by a practitioner.

One of the biggest events in the specialty pharmacy market is happening this year: the availability of biosimilars to AbbVie Inc.’s top-selling Humira (adalimumab). With its Jan. 31 launch, Amgen Inc.’s Amjevita (adalimumab-atto) was the first entrant, and others are expected this summer. But biosimilars of other top biologics also are coming down the pike. AIS Health, a division of MMIT, spoke with a variety of industry experts about what they’re watching in this space in 2023.

AIS Health: What kind of impact will be seen from the Humira biosimilars?

Nicole Kjesbo, Pharm.D., director of clinical program development for Prime Therapeutics LLC: Ideally, we’ll see price competition due to multiple biosimilars of Humira launching in 2023.

Jan. 19: The FDA expanded the label of BeiGene, Ltd.’s Brukinsa (zanubrutinib) to include the treatment of adults with chronic lymphocytic leukemia or small lymphocytic lymphoma. The agency initially approved the Bruton’s tyrosine kinase (BTK) inhibitor on Nov. 14, 2019. The agency granted the application orphan drug designation, and its review used the Assessment Aid. The recommended dose of the capsule is 160 mg twice daily or 320 mg once daily. Drugs.com lists the price of 120 80 mg capsules as more than $15,264.

Jan. 19: The FDA granted accelerated approval to Seagen Inc.’s Tukysa (tucatinib) in combination with trastuzumab for the treatment of adults with RAS wild-type, human epidermal growth factor receptor 2 (HER2)-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. The company says the tyrosine kinase inhibitor is the first FDA-approved treatment in HER2-positive metastatic colorectal cancer. The agency first approved the drug on April 17, 2020. The newest indication had priority review and breakthrough therapy designation. Dosing for the tablet is 300 mg twice daily. Drugs.com lists the price of 60 150 mg tablets as more than $12,389.

While the launches of biosimilars of AbbVie Inc.’s best-selling drug Humira (adalimumab) are arguably the top entrants expected on the U.S. marketplace in 2023, numerous other specialty agents are expected to join them. AIS Health, a division of MMIT, spoke with multiple industry experts on what they’re keeping an eye on in the late-stage pipeline.

AIS Health: Are there any big specialty drugs expected to see patent expiration — and potentially generic or biosimilar competition — in 2023?

Nicole Kjesbo, Pharm.D., director of clinical program development for Prime Therapeutics LLC: Generics are expected to launch in 2023 for [AstraZeneca’s] Iressa (gefitinib), [Jazz Pharmaceuticals plc’s] Xyrem (sodium oxybate), [Novartis Pharmaceuticals Corp.’s] Sandostatin LAR (octreotide acetate) and Thalomid (thalidomide) [from Bristol Myers Squibb unit Celgene Corp.]. Stelara [(ustekinumab) from Johnson & Johnson unit Janssen Biotech, Inc.] (IV/SC) and IV Actemra [[(tocilizumab) from Genentech USA, Inc., a member of the Roche Group] lose their exclusivity in 2023.

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.

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.”