FDA

News Briefs: ICER Says Evidence for Beti-Cel Demonstrates Net Health Benefit

The Institute for Clinical and Economic Review (ICER) unanimously determined that evidence for bluebird bio, Inc.’s betibeglogene autotemcel gene therapy “is adequate to demonstrate that the net health benefit of beti-cel is superior to that of standard clinical management,” it said in a final evidence report published July 19. “Given the high costs of standard care, cost-effectiveness modeling finds beti-cel meets commonly accepted value thresholds at an anticipated price of $2.1 million — if that price is subject to an 80% payback for treatment failure,” stated ICER in a press release. The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) unanimously voted in June in support of approval for the treatment of people with beta thalassemia who require regular red blood cell transfusions. The FDA is expected to make a decision on the application by Aug. 19.

Payers, Dermatologists Say They Are Interested in New Psoriasis Topicals Vtama, Zoryve

Two new drugs to treat plaque psoriasis are adding to the topical options available to treat the condition. Both payers and dermatologists have expressed interest in the agents, according to Zitter Insights, and the class remains a high priority for payer management.

On May 23, the FDA approved Roivant Sciences subsidiary Dermavant Sciences, Inc.’s Vtama (tapinarof) cream for the topical treatment of plaque psoriasis in adults, regardless of disease severity. The company said the agent is the first and only FDA-approved steroid-free topical medication in its class. Dosing of the aryl hydrocarbon receptor agonist is once daily on affected areas, and the drug has no restrictions on length of use. The price for one tube of the drug is $1,325.

News Briefs: Court Ends Patent Thicket Lawsuit Against AbbVie

A U.S. appeals court ruled that AbbVie Inc. does not need to defend itself from a lawsuit brought by the city of Baltimore, unions and insurance carriers that alleges the pharma giant used a patent thicket to improperly protect Humira (adalimumab) from competition. Before the ruling, the suit had the potential to upend widespread pharma industry business practices if it had been decided against AbbVie. It also comes amid news that the Biden administration is launching efforts to prevent patent thicketing, a process by which pharmaceutical companies extend patent exclusivity beyond what patent law ostensibly allows. Two laws, the Hatch-Waxman Act and Orphan Drug Act, set typical patent windows at five years and seven years, respectively. High-level officials at the FDA and the U.S. Patent and Trademark Office in July said they would be working together to scrutinize certain practices that could potentially lead to delays in competition from biosimilars and generics.

Looming FDA Decisions Could Swell Number of Gene Therapies on Market

In its latest drug pipeline report, UnitedHealth Group-owned PBM Optum Rx takes a look at two high-priced gene therapies that are slated for FDA approval soon. But those are far from the only gene therapies that payers should be watching, an Optum Rx executive tells AIS Health.

Both drugs highlighted in Optum’s report, Zynteglo (betibeglogene autotemcel) and Skysona (elivaldogene autotemcel), are manufactured by bluebird bio, Inc., a Massachusetts-based biotech company. An FDA advisory panel in June recommended both drugs for approval. Currently, only two gene therapies are on the U.S. market: Luxturna and Zolgensma.

New FDA Approvals: FDA Expands Patient Population for Diacomit

July 14: The FDA expanded the patient population of Biocodex, Inc.’s Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in people between the ages of 6 months to 2 years and weighing at least 7 kg who are taking clobazam. The agency first approved the treatment on Aug. 20, 2018. The drug is available as a capsule and an oral suspension. Dosing is 50 mg/kg/day for both routes of administration. Drugs.com lists the price of 60 250 mg capsules and 60 250 mg powder for reconstitution as more than $1,589.

July 14: The FDA expanded the label of Pfizer Inc.’s Xalkori (crizotinib) to include the treatment of people at least 1 year old with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The agency initially approved the kinase inhibitor on Aug. 26, 2011. The FDA gave the agent orphan drug designation and granted the application priority review; that review used the Assessment Aid, a voluntary submission from the applicant to assist the FDA in its analysis. Dosing for the newest indication of the capsule in adults is 250 mg twice daily. The recommended pediatric dosage is 280 mg.m2 twice daily based on body surface area. Drugs.com lists the price of 60 250 mg capsules as more than $20,657.

Expanded Label Puts SMA Drugs on Even Terms for Youngest Patients

A recent FDA approval of a label expansion put the three marketed therapies for spinal muscular atrophy (SMA) on equal footing for the youngest patients. A recent survey found that many payers are covering sequential use of the costly agents, including a gene therapy.

The FDA initially approved Evrysdi (risdiplam) from Roche Group member Genentech USA, Inc. on Aug. 7, 2020, for the treatment of SMA in people at least 2 months old. On May 30, 2022, the agency expanded the drug’s label to include the treatment of babies less than 2 months old. The medication is an oral solution administered by mouth or feeding tube and can be administered by a patient or caregiver at home after a recommended consultation with a health care professional prior to the first dose.

Pharma Patent Practices Come Under Scrutiny From Congress, FDA, PTO

As Congress again proposes drug pricing efforts, many of its members, as well as a couple of government agencies, have pharma manufacturers in their crosshairs for a somewhat related reason: their patent processes. The FDA and the U.S. Patent and Trademark Office (USPTO) recently said they would be working together to scrutinize certain practices that could potentially lead to delays in competition from biosimilars and generics.

The move follows President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”

New FDA Approvals: FDA Grants Additional Indication to CellCept

June 6: The FDA expanded the label of Roche Group member Genentech USA, Inc.’s CellCept (mycophenolate mofetil) to include, in combination with other immunosuppressants, prophylaxis of organ rejection in people at least 3 months old who have received an allogenic heart transplant or an allogenic liver transplant. The agency first approved the drug on May 3, 1995. Dosing for the newest uses is based on body surface area and indication. The drug is available as a capsule, tablet, oral suspension and intravenous injectable. Website GoodRx.com lists the price of 60 500 mg tablets as more than $1,070.

June 7: The FDA granted another indication to Sanofi and Regeneron Pharmaceuticals, Inc.’s Dupixent (dupilumab) for the treatment of moderate-to-severe atopic dermatitis in people between the ages of 6 months and 5 years whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The agency initially approved the subcutaneous injectable on March 28, 2017. The FDA gave the new indication priority review. Dosing for people weighing 5 kg to less than 15 kg is 200 mg every four weeks; for those weighing 15 kg to less than 30 kg, dosing is 300 mg every four weeks. The drug’s list price, regardless of dose, is $3,384.83 per carton, which includes either two prefilled pens or two prefilled syringes.

Pharma Patent Practices Come Under Scrutiny From Congress, FDA, PTO

As Congress again proposes drug pricing efforts, many of its members, as well as a couple of government agencies, have pharma manufacturers in their crosshairs for a somewhat related reason: their patent processes. The FDA and the U.S. Patent and Trademark Office (PTO) recently said they would be working together to scrutinize certain practices that could potentially lead to delays in competition from biosimilars and generics.

The move follows President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”

Payers, Dermatologists Say They Are Interested in New Psoriasis Drug Vtama

A new drug to treat plaque psoriasis is the first topical novel chemical entity launched in the U.S. for the condition in 25 years. Both payers and dermatologists have expressed interest in the agent, according to Zitter Insights.

On May 23, the FDA approved Roivant Sciences subsidiary Dermavant Sciences, Inc.’s Vtama (tapinarof) cream for the topical treatment of plaque psoriasis in adults, regardless of disease severity. The company says the agent is the first and only FDA-approved steroid-free topical medication in its class. Dosing of the aryl hydrocarbon receptor agonist is once daily on affected areas, and the drug has no restrictions on length of use. The price for one tube of the drug is $1,325.