Aug. 7: The FDA approved Citius Pharmaceuticals, Inc.’s Lymphir (denileukin diftitox-cxdl) for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one systemic therapy. The immunotherapy is the only CTCL treatment that targets the interleukin-2 (IL-2) receptor found in T-cell lymphomas and regulatory T cells (Tregs). The rare disease is a chronic non-Hodkin lymphoma that primarily affects the skin. The drug is a reformulation of Ontak (denileukin diftitox), which was on the U.S. market from 2008 to 2014, when it was voluntarily withdrawn “to enable manufacturing improvements.” The FDA gave the therapy orphan drug designation. The recommended dose is 9 mcg/kg per day based on actual body weight via a 60-minute intravenous infusion on days one through five of a 21-day cycle. The company says it expects to launch the agent within the next five months.

Commercial and government payers can play a key role in generating real-world evidence (RWE) and helping the health care system reduce costs and improve outcomes, according to a special communication published last month in JAMA. Meanwhile, speakers at a July 25 virtual meeting sponsored by Duke University’s Margolis Institute for Health Policy emphasized that payers should work together with pharmaceutical companies, medical device manufacturers and policymakers to institute better ways to incorporate RWE into practice.

Mark McClellan, M.D., Ph.D., director of the Margolis Institute and a former FDA commissioner and HHS administrator, defined real world data as “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources” such as registries, wearable devices and electronic health records. He added that RWE is “clinical evidence about the use, potential benefits or risks of a medical product or practice or care delivery model that’s derived from the analysis of real-world data.”

July 8: The FDA approved ANI Pharmaceuticals, Inc.’s L-glutamine oral powder to reduce the acute complications of sickle cell disease in people at least 5 years old. The company says it is the first AA-rated approved generic for Emmaus Medical, Inc.’s Endari. Dosing for people weighing less than 30 kilograms is one packet twice daily; for those between 30 and 65 kilograms, dosing is two packets twice daily, and for those more than 65 kilograms, dosing is three packets twice daily. Drugs.com lists the price of 60 packets of Endari as more than $1,505.

June 7: The FDA expanded the treatment area of Almirall, LLC’s Klisyri (tirbanibulin) from up to 25 cm2 of the face or scalp to up to 100 cm2 for the treatment of adults with actinic keratosis. The agency first approved the microtubule inhibitor on Dec. 14, 2020. Dosing is one unit-dose packet on the face or scalp once daily for five consecutive days. Drugs.com lists the price of five packets of the ointment as more than $1,179.

June 10: The FDA gave another indication to Sanofi and Regeneron Pharmaceuticals, Inc.’s Kevzara (sarilumab) for the treatment of people weighing at least 63 kg with active polyarticular juvenile idiopathic arthritis. The agency initially approved the interleukin-6 (IL-6) receptor antagonist on May 22, 2017. The recommended dose is 200 mg via subcutaneous injection every two weeks. Drugs.com lists the price of both the 150 mg/1.14 mL and 200 mg/1.14 mL as more than $4,582.

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.

The FDA recently approved the second gene therapy for hemophilia B, Pfizer Inc.’s Beqvez (fidanacogene elaparvovec-dzkt). While the agent offers an additional treatment option with the potential for freedom from regular infusions of factor therapy, its price — which is equal to that of its competitor — may be too high for many payers, according to a Zitter Insights survey. Industry experts say that it may suffer from some of the challenges other cell and gene therapies have faced in gaining a foothold in the U.S. market.

On April 25, the FDA approved Beqvez for the treatment of adults with moderate to severe hemophilia B who use factor IX (FIX) 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.

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.

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.

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.