With the FDA’s approval of three biosimilars in May, that brings the total number of these drugs to 53 since the agency’s green lighting of Sandoz’s Zarxio (filgrastim-sndz) on March 6, 2015. With all three agents also gaining interchangeability status, that brings the count to 13 biosimilars with this designation. Payer and provider respondents to a Zitter Insights survey said they expect to see increased use in rheumatoid arthritis (RA) and ophthalmic biosimilars, among others, this year, while oncologists cited agents for non-small cell lung cancer (NSCLC) as the area in which they expected to see the most increase.

On May 20, the FDA approved the first biosimilars of Regeneron Pharmaceuticals, Inc.’s Eylea (aflibercept): Biocon Ltd. subsidiary Biocon Biologics Ltd.’s Yesafili (aflibercept-jbvf) and Samsung Bioepis Co., Ltd. and Biogen Inc.’s Opuviz (aflibercept-yszy).

April 30: The FDA expanded the patient population of CSL Vifor’s Mircera (methoxy polyethylene glycol-epoetin beta) to include the treatment of anemia associated with chronic kidney disease in pediatric patients 3 months old to 17 years old on dialysis and not on dialysis who are converting from another erythropoiesis-stimulating agent after their hemoglobin level was stabilized with an ESA. The agency also approved a subcutaneous route of administration for pediatric patients. The FDA first approved the long-acting ESA on Nov. 14, 2007. Dosing for the newest use is once every four weeks based on total weekly epoetin alfa or darbepoetin alfa dose at the time of conversion. The agent is available in both intravenous and subcutaneous formulations, and patients younger than 6 years old should maintain the same route of administration as the previous ESA. Drugs.com lists the price of one 75 mcg/0.3 mL injectable solution as more than $237.

Multiple developments around biosimilar versions of AbbVie Inc.’s Humira (adalimumab) have occurred. Teva Pharmaceuticals, a unit of Teva Pharmaceuticals Industries Ltd., and Alvotech launched Simlandi (adalimumab-ryvk) in the U.S., the companies disclosed on May 20. The FDA approved the interchangeable, high-concentration, citrate-free drug on Feb. 23 for nine of its reference drug’s indications.…Boehringer Ingelheim said on May 13 that it had signed an agreement with Quallent Pharmaceuticals, a private-label pharmaceutical distributor that is a wholly owned subsidiary of The Cigna Group, to offer both high- and low-concentration, citrate-free versions of adalimumab-adbm through a copay assistance program. The tumor necrosis factor (TNF) inhibitor’s interchangeable designation applies to the low-concentration version. Boehringer will continue to commercialize that agent and its branded version, Cyltezo, which has approval for nine of Humira’s indications.…Celltrion USA has made its high-concentration, citrate-free adalimumab-aaty available at a low wholesale acquisition cost that is an 85% discount to Humira’s WAC, the company revealed on May 9. Its branded version, Yuflyma, remains available at a 5% discount to Humira’s WAC. The agent is approved for eight of Humira’s indications.

While health care payers are facing a variety of issues, paying for multimillion-dollar cell and gene therapies (CGTs) is one of the most pressing, as evidenced by sessions at two recent AHIP conferences. Multiple speakers discussed various approaches to the agents, as well as challenges payers need to tackle, but all acknowledged that a truly successful model has yet to be implemented.

Many CGTs are in the pipeline, impacting potentially millions of patients and prompting many questions around affordability and accessibility, stated Sean Dickson, senior vice president of pharmaceutical policy at AHIP, during a session in Baltimore titled “Cell and Gene Therapies: Regulatory Updates and Coverage Policies.” “Oncology is where it will get really interesting,” and these agents will have the greatest impact on Medicare payers.

Optum Rx announced on May 20 a new program, Clear Trend Guarantee, that will combine guarantees into a single per member cost metric. The PBM will make Clear Trend Guarantee available to plan sponsors starting on Jan. 1, 2025. In a press release, Optum Rx described Clear Trend Guarantee as “an alternative, guarantee-based pricing model that combines retail, home delivery, specialty drug and rebate components into one per member guarantee” and said the program would “ultimately deliver value to individuals and families who want predictability in their premiums and drug costs.” Meanwhile, Patrick Conway, Optum Rx’s CEO, wrote in a May 22 LinkedIn post that Clear Trend Guarantee “means plan sponsors will have another option to manage their pharmacy spend with greater predictability and choice to help manage lowest net cost.” Optum Rx last year announced other alternative pricing models, including a pass-through model using average ingredient costs.

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.

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.

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.

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.

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.