Aimmune Therapeutics Inc.'s Palforzia, formerly known as AR-101, is on track to be the first peanut allergy treatment to come to what could be a blockbuster market. While clinical trials showed a higher rate of allergic reactions, dropouts and epinephrine use in the Palforzia arm, FDA briefing documents ahead of the biologic's adcom meeting Friday direct the Allergenic Products Advisory Committee (APAC) to look at reactions to accidental exposures as perhaps a better predictor of real-world utility than a double-blinded placebo-controlled food challenge. Allergic reactions to Palforzia, a peanut allergy powder, are not surprising, Piper Jaffray senior research analyst Chris Raymond said, given "that Palforzia is itself a food challenge and one would expect when exposing an allergic population to allergens, that reactions can occur, as is commonly seen in other therapies green-lighted by APAC. . . . The clinical value of Palforzia lies in a patient's reaction to accidental exposure." And that's what the proposed indication focuses on – reducing the incidence and severity of allergic reactions, including anaphylaxis after accidental exposure to peanut in patients ages 4 through 17. In the phase III PALISADE trial, patients in the placebo arm saw more than a threefold higher incidence in adverse events requiring treatment as a result of accidental exposure than those on Palforzia. Based on his reading of the briefing documents for the adcom, Raymond said he expects a 2020 approval for Palforzia, which has been designated as both a fast track and breakthrough therapy. He also expects the drug to generate sales of more than $1.5 billion by 2025. Investors seemed to agree, given the heavy trading of Brisbane, Calif.-based Aimmune stock (NASDAQ:AIMT) after the FDA released its briefing materials Wednesday. Aimmune closed at $25.46, up 15.4% over Tuesday's close of $22.06. Palforzia had been in a race with DBV Technologies Inc.'s Viaskin peanut allergy vaccine candidate, but the French company withdrew its application last December after the FDA said it lacked sufficient manufacturing data. (See BioWorld, Dec. 21, 2018.)

Whether oral semaglutide offers real value for people with type 2 diabetes will depend on the drug's long-term effectiveness and how Novo Nordisk A/S prices the drug if the FDA approves it, the Institute for Clinical and Economic Review (ICER) concluded in the draft evidence report it released Wednesday. ICER's assessment determined that oral semaglutide likely would produce incremental benefit vs. alternative treatments in terms of preventing major adverse cardiovascular events (MACE). But given the complexity of type 2 diabetes, its large number of co-morbidities and patient-specific clinical management, "MACE prevention is only part of the treatment puzzle, and other treatments may provide better overall benefit and at lower cost," according to the report. "Based on the current clinical evidence, with limited follow-up and without knowing the eventual price for oral semaglutide, we are unable to draw conclusions on its cost effectiveness with any certainty." In the assessment, ICER compared oral semaglutide with Victoza (liraglutide), another Novo Nordisk GLP-1 agonist; Boehringer Ingelheim International GmbH and Eli Lilly and Co.'s Jardiance (empagliflozin), an SGLT-2 inhibitor; and Merck & Co. Inc.'s Januvia (sitagliptin), a DPP-4 inhibitor. The FDA is expected to make a decision this month on the use of oral semaglutide to control blood glucose in patients with type 2 diabetes. A decision on a second indication – a reduction in MACE in adults with type 2 diabetes and established cardiovascular disease – is expected in January. Novo Nordisk's injectable form of the drug, marketed as Ozempic, was approved by the FDA in 2017. If approved, oral semaglutide would be the first oral formulation of a GLP-1 receptor agonist to become available. Comments on the draft evidence report are due by Oct. 8. ICER plans to publish its final report on oral semaglutide Oct. 31.

The U.K.'s Medicines and Healthcare Products Regulatory Agency (MHRA) is seeking the public's advice on ways to ensure the patient voice is heard in licensing new drugs and identifying safety issues with drugs or medical devices. As part of a public consultation on how it engages with and involves patients in its work, the MHRA will hold a series of public events beginning next month and is asking the public to respond to an online questionnaire concerning the agency's current communication practices and suggestions for improvement.

Redback SARMs, an Australian business that marketed selective androgen receptor modulators (SARMs), paid $15,210 in penalties after the Therapeutic Goods Administration (TGA) slapped it with six infringement notices for the alleged advertising of prescription-only substances, including SARMs and melanotan II. In announcing the penalties, the TGA reminded businesses that prescription drugs cannot be advertised to the public. Promoting general classes of prescription drugs, such as SARMs, is generally considered advertising, the TGA said, as are online sites that allow consumers to review and select prescription drugs.

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