Drug Approvals

FDA Approves Second Gene Therapy, but Will Price Prove to Be Too High?

The FDA recently approved the second gene therapy for hemophilia B, Pfizer Inc.’s Beqvez (fidanacogene elaparvovec-dzkt). But while the manufacturer priced the agent at parity to the other treatment, that price may still be too high for many payers, according to a Zitter Insights survey.

On April 25, the FDA approved Beqvez for the treatment of adults with moderate to severe hemophilia B who use factor IX prophylaxis therapy; have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes and do not have neutralizing antibodies to adeno-associated virus (AAV) serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test. The manufacturer launched a warranty program for the intravenous infusion based on durability of patient response to treatment.

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Established TKI Gains New Indication to Treat Aggressive Cancer

After first gaining approval more than a decade ago, Takeda Pharmaceuticals U.S.A., Inc.’s Iclusig (ponatinib) recently gained approval for the frontline treatment of an aggressive blood cancer. One clinical trial found that people on the agent experienced complete remission more than twice as often as those on a comparator therapy. Industry sources point to the drug’s clinical efficacy as a significant development in the treatment of the disease.

On March 19, the FDA gave accelerated approval to Iclusig in combination with chemotherapy for the treatment of adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The newest application had priority review and orphan drug designation, and its review used the Real-Time Oncology Review and the Assessment Aid.

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New FDA Approvals: FDA Approves AstraZeneca’s Voydeya

March 29: The FDA approved Alexion, AstraZeneca Rare Disease’s, Voydeya (danicopan) as an add-on therapy to ravulizumab (currently available as the company’s Ultomiris) or eculizumab (currently available as the company’s Soliris) for the treatment of extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria. The agent is a first-in-class factor D inhibitor, and it has breakthrough therapy designation. Initial dosing for the tablet is 150 mg three times a day, which can be increased to 200 mg three times a day. Drugs.com lists the price of 180 50 mg-100 mg tablets as more than $4,359.

April 5: The FDA expanded the patient population of AstraZeneca’s Fasenra (benralizumab) to include the add-on maintenance treatment of people aged 6 to 11 with severe asthma with an eosinophilic phenotype. The agency first approved the interleukin-5 receptor alpha-directed cytolytic monoclonal antibody on Nov. 14, 2017. Dosing for the newest indication for pediatric patients weighing less than 35 kg is 10 mg via subcutaneous injection every four weeks for the first three doses and then once every eight weeks; for those at least 35 kg, dosing is 30 mg every four weeks, followed by once every eight weeks. The list price for one 30 mg/mL solution is $5,511.41.

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High Price Tag, Accelerated Approval for New NASH Drug May Give Payers Pause

When the FDA approved Rezdiffra (resmetirom) in March, it gave patients with a serious form of liver disease a long-awaited treatment option tailored to their specific condition. But the drug’s accelerated approval and high price tag are already spurring at least one health insurer to consider options like restrictive coverage criteria and value-based contracting.

NASH represents a progression from a more common condition, nonalcoholic fatty liver disease (NAFLD). It occurs when patients start developing liver inflammation that can eventually lead to liver scarring, dysfunction, and even liver failure and cancer.

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News Briefs: FDA Sets Date for Donanemab Panel

The FDA on June 10 will consult a panel of outside experts on donanemab, Eli Lilly & Co.’s Alzheimer’s drug. In the advisory committee hearing, third-party experts in neuroscience and drug development will hear presentations from the FDA and Lilly, then be able to question Lilly and FDA officials on the data behind donanemab’s application for approval. A decision on the drug had been expected earlier this year, but the FDA decided to delay it in order to let the advisory committee hearing take place. However, the advisory committee’s recommendation does not carry any official weight. That was a point of controversy after the FDA in 2021 granted accelerated approval to Biogen Inc. & Eisai’s Co.’s Aduhelm (aducanumab), ignoring near-unanimous disapproval from a similar panel. Several advisers resigned from the advisory panel after the Aduhelm accelerated approval decision.

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Rumored Regs Could Help Payers, PBMs Streamline Digital Therapeutic Coverage

Notable changes to the way breakthrough medical devices are covered by Medicare and other payers could be coming soon, with CMS poised to make some therapeutics eligible for Medicare coverage, and some stakeholders pushing for new legislation to expand digital therapeutic coverage. In the meantime, commercial health plans and PBMs are grappling with how best to cover prescription digital therapeutics (PDTs).

Health plans and PBMs have varying approaches to digital therapeutic reimbursement. Some plans may place one digital therapeutic in the medical benefit, while others may cover PDTs as part of a pharmacy benefit. That’s because PDTs occupy a unique space in the health benefits landscape: They aren’t pharmaceuticals, but they share many characteristics of a maintenance medication in practice.

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Rumored Regs Could Help Payers, PBMs Streamline Digital Therapeutic Coverage

Notable changes to the way breakthrough medical devices are covered by Medicare and other payers could be coming soon, with CMS poised to make some therapeutics eligible for Medicare coverage, and some stakeholders pushing for new legislation to expand digital therapeutic coverage. In the meantime, commercial health plans and PBMs are grappling with how best to cover prescription digital therapeutics (PDTs).

Health plans and PBMs have varying approaches to digital therapeutic reimbursement. Some plans may place one digital therapeutic in the medical benefit, while others may cover PDTs as part of a pharmacy benefit. That’s because PDTs occupy a unique space in the health benefits landscape: They aren’t pharmaceuticals, but they share many characteristics of a maintenance medication in practice.

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Older Drug Gets New Indication for Use in Aggressive Cancer

A drug that the FDA first approved more than a decade ago was recently approved for the first-line treatment of an aggressive blood cancer. One clinical trial showed that people on the agent experienced complete remission more than twice as often as those on a comparator therapy.

On March 19, the FDA gave accelerated approval to Takeda Pharmaceuticals U.S.A., Inc.’s Iclusig (ponatinib) in combination with chemotherapy for the treatment of adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). The newest application had priority review and orphan drug designation, and its review used the Real-Time Oncology Review and the Assessment Aid.

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Budgets Propose Eliminating Interchangeability Status for Biosimilars

When then-President Barack Obama signed the Affordable Care Act (ACA) into law on March 23, 2010, he established the 351(k) biosimilar pathway via the Biologics Price Competition and Innovation Act (BPCIA), which amended the Public Health Service (PHS) Act. Since then, the FDA has approved almost 50 biosimilars, with about one-fifth of those gaining interchangeable status. That distinction, however, has been increasingly under fire, most recently in President Biden’s fiscal year (FY) 2025 budget, which proposes eliminating the interchangeability designation entirely. That could help boost uptake of biosimilars, resulting in prescription drug savings, say some industry experts.

In contrast to the EU, whose European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) clarified in September 2022 that all biosimilars approved in the EU are interchangeable, the FDA has created two levels of biosimilars: biosimilars and interchangeable biosimilars.

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New FDA Approvals: FDA Approves BeiGene’s Tevimbra

March 13: The FDA approved BeiGene, Ltd.’s Tevimbra (tislelizumab-jsgr) for the treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma after prior systemic chemotherapy that did not include a programmed death receptor-1 (PD-1)/programmed death-ligand 1 (PD-L1) inhibitor. Recommended dosing for the PD-1 inhibitor is 200 mg via intravenous infusion once every three weeks. The initial dose is administered over 60 minutes; if it is tolerated, subsequent doses can be administered over 30 minutes. The drug will be available in the second half of 2024.

March 13: The FDA gave an additional indication to Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) for the treatment of cholestatic pruritus in people at least 5 years old with progressive familial intrahepatic cholestasis (PFIC). The agency first approved the ileal bile acid transporter inhibitor on Sept. 29, 2021. The new indication has orphan drug designation, as well as breakthrough therapy designation for PFIC type 2. The starting dose for the newest use of the oral solution is 285 mcg/kg once daily in the morning, increasing to 285 mcg/kg twice daily, then 428 mcg/kg twice daily and then 570 mcg/kg twice daily. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $56,239.

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