Specialty Drugs

New First-in-Class Therapy Should Help Ease Disease Burden for People With Follicular Lymphoma

The FDA recently approved a new first-in-class agent for follicular lymphoma, an important development for people with the condition, according to one oncologist. Payers and oncologists both agreed that the drug’s approval should help ease the disease burden somewhat for people suffering from the condition, according to a Zitter Insights survey.

On Dec. 22, the FDA gave accelerated approval to Roche Group member Genentech USA, Inc.’s Lunsumio (mosunetuzumab-axgb) for the treatment of adults with relapsed or refractory follicular lymphoma after at least two lines of systemic therapy. The drug is a first-in-class CD20xCD3 T-cell engaging bispecific antibody. Dosing for the agent — which can be done in an outpatient setting — is 1 mg via intravenous infusion on day one of cycle one, 2 mg on day eight of cycle one and 60 mg on day 15 of cycle one, each over a minimum of four hours. On day one of cycle two, dosing is 60 mg, and then in cycle three and following cycles, dosing is 30 mg on day one; administration can be reduced to two hours if cycle one infusions were tolerated. Dosing for eight cycles is recommended. If people have a partial response or stable disease after eight cycles, an additional nine cycles may be administered. The drug’s price for eight cycles is about $180,000.

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People Are Seeing Benefits From Biomarker Testing, but Barriers to Coverage Remain

Biomarker testing is an important tool in cancer care, but a recent survey found payer coverage issues are creating access barriers. According to CancerCare, researchers found that biomarkers helped providers offer personalized care for various cancers for nearly all — 93% — respondents. Twenty percent of surveyed patients were able to forgo unneeded chemotherapy and/or radiation, while 10% found that they were eligible for a clinical trial.

However, the survey also found that 29% of people who had biomarker testing had insurance that did not cover it, prompting them to undergo appeals, obtain financial assistance or pay out of pocket for the service. In addition, 25% of patients said that their insurer required prior authorization (PA) for the process, a tactic that can delay access to treatment.

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New FDA Approvals: FDA Approves Acadia’s Daybue for Rett Syndrome

March 10: The FDA approved Acadia Pharmaceuticals Inc.’s Daybue (trofinetide) for the treatment of Rett syndrome in people at least 2 years old. The drug is the only FDA-approved treatment for the complex, rare neurodevelopmental disorder, which is estimated to affect 6,000 to 9,000 people in the U.S. The agency gave the drug fast track status and orphan drug designation; it also gave the company a rare pediatric disease priority review voucher. Dosing of the oral solution is weight based and can be given orally or via gastrostomy tube. The company has not disclosed the agent’s price, but analyst estimates are between $400,000 and $600,000 per year. Acadia said it expects the agent to be available by the end of April.

March 13: The FDA expanded the patient population of Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) to include the treatment of cholestatic pruritus in people with Alagille syndrome who are at least 3 months old. The agency initially approved the oral solution on Sept. 29, 2021. The starting dose is 190 mcg/kg once daily and then increased to 380 mcg/kg after one week. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters as more than $53,712.

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Bladder Cancer Gene Therapy Adstiladrin Offers New Option in Slim Category

In late 2022, the FDA 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 expect to manage the drug to label. While the drug gives another treatment alternative to a much-needed area, it likely will continue to put financial pressure on payers, industry experts tell AIS Health, a division of MMIT.

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.

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Makena Withdrawal Brings End to Drug at Times Mired in Controversy

The FDA will withdraw Makena (hydroxyprogesterone caproate) from the U.S. market, the agency said April 6. The move follows manufacturer Covis Pharma Group saying on March 7 that it had requested an “orderly wind-down” of the drug from the market. However, “effective today, Makena and its generics are no longer approved and cannot lawfully be distributed in interstate commerce,” said the agency in its statement. “Approvals of these drugs have been withdrawn because the drugs are no longer shown to be effective and the benefits do not outweigh the risks for the indication for which they were approved.”

The decision brings to a close a long-running saga that started more than a decade ago, as well as shines a spotlight on the FDA’s often-criticized accelerated approval pathway.

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Prime, Magellan Studies Reveal Ways to Squeeze More Value From Specialty Drugs

Prime Therapeutics LLC and Magellan Rx Management, a Prime company as of the end of last year, recently presented findings from a series of studies that used integrated medical and pharmacy claims to assess real-world drug use. Their findings show that different strategies can help identify potential member issues that could impact payer costs.

The studies were presented at the Academy of Managed Care Pharmacy (AMCP) Annual Meeting, held in San Antonio, Texas, March 21 to 24.

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News Briefs: J&J, AbbVie Units Will Withdraw Two Imbruvica Indications

The Janssen Pharmaceutical Companies of Johnson & Johnson and AbbVie Inc. unit Pharmacyclics LLC are voluntarily withdrawing two indications for Imbruvica (ibrutinib) that had accelerated approval, the companies said on April 6. Following consultation with the FDA, the companies will remove two non-Hodgkin’s lymphoma indications from the drug’s label: the treatment of people with mantle cell lymphoma (MCL) who have received at least one prior therapy, and the treatment of marginal zone lymphoma (MZL) in people who require systemic therapy and have received at least one anti-CD20-based therapy. The decision follows discussion of the results of two Phase III studies — SHINE for MCL and SELENE for MZL — with the FDA, which “advised that the primary outcomes…were considered insufficient to support conversion to full approval.” The Bruton’s tyrosine kinase inhibitor’s other FDA-approved indications, which comprise four disease areas, including three hematologic cancers, are not impacted. The agency first approved the drug on Nov. 13, 2013, when it granted accelerated approval for the MCL indication. It gave accelerated approval for MZL use on Jan. 19, 2017. Since 2020, more than 20 oncology indications and/or drugs approved through the accelerated pathway have been withdrawn.

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Coverage of Certain Cancer Screenings, PrEP Are at Risk After Ruling

A judge recently struck down certain preventive services coverage mandated by the Affordable Care Act (ACA). If upheld, the decision could upend access to potentially life-saving services that have been provided for free for more than a decade.

Judge Reed O’Connor from the U.S. District Court for the Northern District of Texas issued the ruling in Braidwood Management v. Becerra (No. 4:20-cv-00283-O) on March 30. The following day, the Justice Department said it was appealing the decision, sending the case to the U.S. Court of Appeals for the Fifth Circuit, potentially reaching the Supreme Court.

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Patient-Paid Prescriptions Are Challenging Traditional Industry Model

As the use of patient-paid prescriptions continues to grow, it is posing a challenge to the pharmacy benefit market, contended longtime industry expert Adam J. Fein, Ph.D., CEO of Drug Channels Institute, during a recent webinar. And with some industry developments such as the Inflation Reduction Act likely to impact the uptake of high-price/high-rebate drugs, patient-paid prescriptions could become an even bigger disruption within the market than they already are.

The issue of patient-paid prescriptions — which include both cash-pay claims and discount-card claims — is “potentially something that’s going to actually be genuinely disruptive, actually something that could change the structure of both the PBM industry and how plan sponsors” think about benefit design, Fein asserted. Both approaches, he explained, remove the third-party payer, allowing patients to make a transaction directly with a pharmacy.

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Point32Health Exec Says Outcomes-Based Pacts Can Unite Payers, Pharma

While drugs are increasingly hitting the market that address unmet needs and even offer cures for some rare diseases, private insurers are highly concerned about such therapies’ eye-popping price tags, a recent survey indicated. But one prominent payer executive who spoke during AHIP’s Medicare, Medicaid, Duals & Commercial Markets Forum suggested that insurers are better off working collaboratively with drugmakers to ensure prices are tied to value — rather than engaging in an inter-industry war of words.

“More and more we’re seeing drugs come through with limited evidence through accelerated approval processes, which generally is a marker for an unmet need, which is a good thing. But the evidence can be thin,” said Michael Sherman, M.D., executive vice president and chief medical officer of Point32Health. During a March 14 panel at the AHIP forum, Sherman pointed to the example of Makena (hydroxyprogesterone caproate), a drug that aims to reduce preterm births but failed to prove clinical effectiveness in trials conducted after it received accelerated approval. With the FDA poised to make a final decision on the drug’s status, Clovis Pharma Group recently announced it would voluntarily pull Makena off the market.

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