FDA Action Alert: Theratechnologies, Alnylam, scPharmaceuticals and More

2022-10-03

抗体优先审批引进/卖出

Sarah Silbiger/Getty Images The FDA has a relatively light calendar for October with Theratechnologies' Trogarzo for HIV, Alnylam's Lumasiran for advanced primary hyperoxaluria, scPharmaceuticals' Furoscix for heart failure, and more. Here's a look at the agency's schedule for the month. Theratechnologies' Trogarzo for HIV Theratechnologies has a target action date of Oct. 3 for its supplemental Biologics License Application (sBLA) for an IV Push formulation of Trogarzo for HIV. Trogarzo is a CD4-directed post-attachment HIV-1 inhibitor. The new formulation is more convenient. It can allow an infusion within 30 seconds without dilution compared to the 15-minute infusion time of the original IV formulation. The submission was based on results from the TMB-302 Phase III trial, which Theratechnologies' partner, TaiMed Biologics, ran. The study hit the primary endpoint with a 90% confidence interval of the ratio of IV Push to IV Infusion with no serious adverse events. It also hit two secondary endpoints, demonstrating no difference in HIV-1 viral load from the change to IV Push and no detection of anti-Trogarzo antibodies. Alnylam's Lumasiran for Advanced Primary Hyperoxaluria Type 1 Alnylam Pharmaceuticals has a target action date of Oct. 6 for its sNDA for lumasiran to reduce plasma oxalate for patients with advanced primary hyperoxaluria type 1 (PH1). Under the brand name Oxlumo, the drug was approved by the FDA in 2020 for treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients. The sNDA was based on positive six-month data from the ILLUMINATE-C trial, which demonstrated the drug substantially decreased plasma oxalate levels in patients with compromised renal function from PH1, including patients on hemodialysis. scPharmaceuticals' Furoscix for Worsening Heart Failure scPharmaceuticals has a target action date of Oct. 8 for the resubmission of its NDA for Furoscix. The drug is a proprietary formulation of furosemide delivered by an on-body infusor to treat congestion in patients with worsening heart failure. The drug is formulated to a neutral pH, which allows for subcutaneous infusion via a wearable pre-programmed on-body drug delivery system allowing for outpatient self-administration. "The resubmission and acceptance of our Furoscix NDA was a significant achievement for our company and potentially gives us line-of-sight to transitioning to a commercial-stage company," stated John Tucker, CEO of scPharmaceuticals. "We believe the NDA addresses all concerns and questions raised in the Complete Response Letter that we received from the FDA in December 2020. As a result, we are preparing for a commercial launch of FUROSCIX, if approved, in the fourth quarter of this year." Harrow's AMP-100 for Eye Anesthesia and Pain Management Harrow Health had a target action date of Oct. 16 for its NDA for AMP-100. The drug was being considered for ocular surface anesthesia and intraoperative pain management during ocular surgery. On Sept. 27, the FDA approved it for that indication under the brand name Iheezo (chloroprocaine hydrochloride ophthalmic gel) 3%. Harrow Health acquired the drug from Sintetica in July 2021. The NDA was based on data from a Phase III trial that successfully compared its efficacy and tolerability to current standards of care. "We have always believed in the unique clinical value of Iheezo, and now that Iheezo is approved for use in the U.S. market, it has the potential to become an indispensable premium tool for eyecare professionals and their patients requiring ocular surface anesthesia," stated Mark L. Baum, chairman and CEO of Harrow. Ipsen's Palovarotene for Heterotopic Ossification with Fibrodysplasia Ossificans Progressiva The FDA's Endocrinologic and Metabolic Drugs Advisory Committee is meeting on Oct. 31 to discuss Ipsen Biopharmaceuticals' NDA for palovarotene capsules. The proposed indications are for the prevention of heterotopic ossification in adults and children, specifically females eight years and older and males 10 years and older, with fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare genetic disorder that results in bone forming where it shouldn't, such as outside of the skeleton and in the soft and connective tissues like the muscles, tendons and ligaments. Bones forming outside the skeleton are called heterotopic ossification. The FDA has assigned a target action date of Dec. 29 for the application after the company resubmitted the NDA for Priority Review. The NDA was originally accepted on May 28, 2021; then the company withdrew it on Aug. 13, 2021.