Cell and Gene Therapies

News Briefs: ACA Marketplace Enrollment Tops 16.3M

During the open enrollment period that ended on Jan. 15 in most states, 16,306,448 people selected an Affordable Care Act marketplace plan, the Biden administration said on Jan. 25. That total represents a 13% increase compared to the same time last year, and accounts for plan selections through Jan. 15 on the 33 states using HealthCare.gov and through Jan. 14 or 15 on the 18 state-based marketplaces. While record-breaking for the exchanges, the signup total for 2023 is not final, as open enrollment continued through Jan. 23 for Massachusetts Health Connector and through Jan. 31 for DC Health Link, Covered California, Get Covered New Jersey, New York State of Health and Health Source Rhode Island. Still, the Biden administration hailed the new enrollment figures, with CMS Administrator Chiquita Brooks-LaSure stating: “On the tenth anniversary of the ACA Marketplaces, the numbers speak for themselves: more people signed up for plans this year than ever before, and the uninsured rate is at an all-time low.”

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New Organization Will Focus on Medical Benefit Drugs

A group of Blue Cross and Blue Shield-affiliated companies recently unveiled a new medication contracting organization focused on medical benefit drugs. The new company, known as Synergie Medication Collective, will be successful in improving the affordability of these treatments and patients’ access to them, an industry expert says, but it also will need to show that patients are seeing those savings.

Unveiled Jan. 5, the company says it “is focused on improving affordability and access to costly medical benefit drugs — ones that are injected or infused by a health care professional in a clinical setting — for nearly 100 million Americans.” It will focus not only on infusible treatments for conditions such as cancer but also on multimillion dollar gene therapies. The company says its goal is to “significantly reduce medical benefit drug costs by establishing a more efficient contracting model based upon its collective reach and engagement with pharmaceutical manufacturers and other industry stakeholders.” It plans to “bring to market several new product offerings” this year, among them “transformative value-based models.”

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New FDA Approvals: FDA Gives Accelerated Approval to Mirati’s Krazati

Dec. 12: The FDA gave accelerated approval to Mirati Therapeutics, Inc.’s Krazati (adagrasib) for the treatment of adults with KRASG12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test (see below brief), who have received at least one systemic therapy. Dosing for the tablet is 600 mg twice daily. The drug is priced at $19,750 per month.

Dec. 12: The FDA approved two companion diagnostics for Krazati (see above brief): Agilent Technologies Inc.’s Agilent Resolution ctDx FIRST assay and Qiagen N.V.’s Qiagen therascreen KRAS RGQ PCR kit. A liquid biopsy next-generation sequencing assay, the Agilent test detects genomic alterations in circulating tumor DNA from plasma. The Qiagen test, first approved July 6, 2012, is a tissue-based assay.

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FDA Approved Four New Gene Therapies, Other Novel Agents in 2022

While the FDA may not have approved the most drugs in a year in 2022, it still gave the green light to a number of agents, many of them specialty medications, as well as granted additional indications to existing therapies. The FDA’s Center for Drug Evaluation and Research (CDER) approved 39 new molecular entities in 2022, and the Center for Biologics Evaluation and Research (CBER) approved 12 biologic license applications, including four new gene therapies in the second half of the year. AIS Health, a division of MMIT, asked some industry sources what the most notable 2022 FDA approvals were and why they were so important.

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New Organization Will Focus on Medical Benefit Drugs

A group of Blue Cross and Blue Shield-affiliated companies recently unveiled a new medication contracting organization focused on medical benefit drugs. The new company, known as Synergie Medication Collective, will be successful in improving the affordability of these treatments and patients’ access to them, an industry expert says, but it also will need to show that patients are seeing those savings.

Unveiled Jan. 5, the company says it “is focused on improving affordability and access to costly medical benefit drugs — ones that are injected or infused by a health care professional in a clinical setting — for nearly 100 million Americans.” It will focus not only on infusible treatments for conditions such as cancer but also on multimillion dollar gene therapies. The company says its goal is to “significantly reduce medical benefit drug costs by establishing a more efficient contracting model based upon its collective reach and engagement with pharmaceutical manufacturers and other industry stakeholders.” It plans to “bring to market several new product offerings” this year, among them “transformative value-based models.”

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2022 Saw Some Pharma Challenges, but Industry Has Continued to Innovate With Novel New Agents

As the pharma industry dealt with the ongoing COVID-19 pandemic amid heightened economic pressures, it largely weathered the storm that was 2022. But with provisions of the Inflation Reduction Act (IRA) set to start rolling out next year, many questions remain that may impact pharma manufacturers. The industry, however, saw continued innovation in novel therapeutics, and in the second half of the year, the number of gene therapies on the U.S. market more than doubled with the approval of three new agents. And while the merger and acquisition (M&A) activity may have been a bit muted compared with past years, 2022 is closing out with the unveiling of the biggest biotech deal of the year: Amgen Inc.’s agreement to purchase Horizon Therapeutics plc for $27.8 billion. AIS Health, a division of MMIT, spoke to industry experts about other 2022 pharma trends.

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FDA Approves Hemophilia B Gene Therapy, but Its Cost May Pose Access Problems for Payers

The FDA recently approved the fifth gene therapy to gain FDA approval, with three of those decisions coming in the second half of this year. While payer respondents to a Zitter Insights survey have expressed interest in the drug, its price may prove to be an obstacle to coverage.

On Nov. 22, the FDA approved uniQure N.V.’s Hemgenix (etranacogene dezaparvovec-drlb) for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. CSL Behring LLC, a CSL business, will market the gene therapy. The agency gave the first-in-class adeno-associated virus A5-based gene therapy — which previously was known as EtranaDez — priority review and orphan and breakthrough therapy designations.

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New FDA Approvals: FDA Issues Emergency Use Authorization to Kineret

Nov. 9: The FDA issued an emergency use authorization to Amgen Inc.’s Kineret (anakinra) for the treatment of COVID-19 in hospitalized adults with pneumonia requiring supplemental oxygen (low- or high-flow oxygen) who are at risk of progressing to severe respiratory failure and likely to have an elevated plasma soluble urokinase plasminogen activator receptor (suPAR). The agency initially approved the interleukin-1 receptor antagonist (IL-1Ra) on Nov. 14, 2001. The recommended dosing for this latest use is 100 mg administered daily by subcutaneous injection for 10 days. Drugs.com lists the price of one 100 mg prefilled syringe as more than $1,265.

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Cell and Gene Therapies Hold Promise, but Stakeholders Must Overcome Challenges to Meet Their Full Potential

Researchers continue to make progress in developing cell and gene therapies that offer the promise of slowing a disease’s progression and even offering a potential cure to patients. And while these agents may offer hope to patients, some challenges exist, including access to the treatments. In order for these products to reach their full potential, stakeholders must work together to overcome these potential barriers.

With its Feb. 28 FDA approval, the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.’s Carvykti (ciltacabtagene autoleucel or cilta-cel) became the sixth chimeric antigen receptor T-cell (CAR-T) therapy approved in the U.S. In addition, the existing CAR-Ts continue to get additional FDA-approved indications added to their labels, including for use in earlier line settings. And in August and September alone, the FDA approved two bluebird bio, Inc. gene therapies: Zynteglo (betibeglogene autotemcel or beti-cel) and Skysona (elivaldogene autotemcel or eli-cel).

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Industry Expert Maintains That Specialty Pharmacy Market Has Reached ‘Inflection Point’

Multiple trends are occurring within the specialty pharmacy industry that could have a huge impact on the space, maintained longtime industry expert Adam J. Fein, Ph.D., CEO of Drug Channels Institute, during a recent webinar. Those include greater competition among specialty products, a slowing of payer spending on specialty drugs and more vertical integration among both payers and providers. “We’re at kind of an inflection point in the specialty market,” he contended during the July 29 webinar, titled Specialty Drugs Update: Trends, Controversies, and Outlook.

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