September 2022 decisions expected from the FDA

Your monthly synopsis of new drugs expected to hit the market

August 15, 2022
At Prime Therapeutics (Prime), we’ve positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs likely to be approved by the U.S. Food and Drug Administration (FDA).

Drug pipeline for September 2022:

9/9/2022: Rolontis® (eflapegrastim)
The FDA is reviewing Spectrum Pharmaceuticals and Hanmi Pharmaceutical’s Rolontis as a long-acting granulocyte colony-stimulating factor (GM-CSF). Rolontis uses Hanmi’s lapscovery technology for treatment of chemotherapy-induced neutropenia. Rolontis is a health care-administered prefilled subcutaneous injection. Rolontis’ application is based on the Phase 3 ADVANCE trial that achieved an absolute risk reduction in severe neutropenia of 8.5 percentage points compared with Amgen’s Neulasta® (84.2% versus 75.7%).1 ADVANCE demonstrated Rolontis was non-inferior in reducing the duration of severe chemotherapy-induced neutropenia in comparison with Neulasta. Phase 3 clinical trial RECOVER also demonstrated Rolontis to be non-inferior to Neulasta in reduction of duration of severe neutropenia in patients with early-stage breast cancer receiving chemotherapy.

 9/10/2022: deucravacitinib
The FDA is reviewing Bristol Myers Squibb’s deucravacitinib, a selective tyrosine kinase 2 (TYK2) inhibitor for oral treatment of adults with moderate to severe plaque psoriasis. Deucravacitinib is seeking approval based on the Phase 3 POETYK PSO clinical trial that evaluated once daily deucravacitinib compared to either placebo or Otezla. Results showed that by 16 weeks, 58.7% of patients who received deucravacitinib achieved a PASI 75 response compared with 35.1% of apremilast and 12.7% placebo.2 Similar products include Amgen’s Otezla® (apremilast), which is currently the only approved oral treatment for all severities of psoriasis.

9/13/2022: linzagolix
ObsEva’s linzagolix is being evaluated by the FDA for treatment of moderate to severe endometriosis-associated pain (EAP). Linzagolix is an oral GnRH receptor antagonist being developed in two regimens: 100mg without hormonal add-back therapy (ABT) or 200mg in combination with ABT. Linzagolix is seeking approval based on the Phase 3 clinical trial EDELWEISS, which met co-primary endpoints of reduction in dysmenorrhea (DYS) and non-menstrual pelvic pain (NMPP) at three months. Results demonstrated rapid reductions in DYS and NMPP compared to placebo (following one and two months of treatment, respectively), as well as sustained reduction up to six months of treatment.3 Similar products include Myovant Sciences’ Orgovyx® (relugolix).

9/16/2022: elivaldogene autotemcel
The FDA is evaluating Bluebird Bio’s elivaldogene autotemcel for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor. Elivaldogene autotemcel utilizes a lentiviral vector (LVV) to add functional ABCD1 gene copies into hematopoietic stem cells. The application for elivaldogene autotemcel is supported by efficacy and safety data from the completed Phase 2/3 STARBEAM study. In clinical studies, patients treated with eli-cel were more likely to achieve both overall and event-free survival than allo-HSCT patients without a matched sibling donor, with the clearest benefit for patients without a matched donor of any type.4 The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously endorsed elivaldogene autotemcel 15 to 0 on the question of whether the benefits outweigh the risks, for the treatment of any subpopulation of children with early active cerebral adrenoleukodystrophy.4 Similar products include allo-HSCT.

9/18/2022: HTX-019 (aprepitant)
Heron Therapeutics’ HTX-019 is seeking FDA approval for prevention of postoperative nausea and vomiting (PONV) in adults. HTX-019 is an intravenous formulation of aprepitant which is a neurokinin-1 (NK1) receptor antagonist.5 HTX-019 is seeking approval via the 505(b)2 pathway using Merck Sharp & Dohme Corp’s Emend® as its reference drug.

9/2022: Rebyota (RBX2660-fecal microbiota transplant)
The FDA is evaluating Ferring/Rebiotix’s Rebyota for treatment of recurrent Clostridioides difficile (C. diff) infection.  Rebyota is a first-in-class microbiota-based live biotherapeutic. Based on the Phase 3 trial, PUNCH CD3, Rebyota successfully met its primary endpoint by demonstrating superior efficacy compared to placebo (70.4% and 58.1%, respectively) at eight weeks post treatment. In addition to these outcomes, Rebyota provided a relative reduction of recurrence of 29.4% compared to placebo.6 Similar products include fecal microbiome transplant.

9/23/2022: Pedmark® (sodium thiosulfate, STS)
Fennec Pharmaceuticals’ Pedmark is being reviewed by the FDA as an intravenous (IV) formulation of the thiol compound that acts as a chemical reducing agent for prevention of ototoxicity induced by cisplatin chemotherapy in pediatric patients one month to less than 18 years of age with localized, non-metastatic solid tumors. Pedmark is seeking approval based on two Phase 3 clinical studies, ACCL0431 and SIOPEL 6. ACCL0431 demonstrated hearing loss that was found to be significantly lower in the Pedmark group compared to cisplatin alone group after adjusting for stratification variables (28.6% Pedmark compared to 56.4% in control group). SIPEL 6 demonstrated hearing loss was significantly less with Pedmark compared to cisplatin alone (33% compared to 63%) which demonstrated a 48% lower incidence of hearing loss. Additionally, the three-year rate of event-free survival was 82% for the cisplatin-sodium thiosulfate group and 79% for the cisplatin alone group; overall survival was 98% and 92%, respectively.9 Similar products include generic STS.

9/29/2022: Albrioza® (sodium phenylbutyrate and taurursodiol)
The FDA is reviewing Amylyx Pharmaceuticals’ Albrioza for oral treatment of amyotrophic lateral sclerosis (ALS). Albrioza application is supported by data from the placebo-controlled Phase 2 CENTAUR trial, along with the open-label extension study. Albrioza’s CENTAUR results were statistically significant in slowing of functional decline, measured by the ALS Functional Rating Scale (ALSFRS-R). The mean rate of decline on ALSFRS-R was 1.24 points per month with Albrioza, as compared with 1.66 points each month with placebo. After 24 weeks of treatment, mean ALSFRS-R scores were significantly higher by 2.92 points among Albrioza-treated patients than those on placebo.8 Additionally, in a survival analysis conducted from the CENTAUR trial patients treated with Albrioza demonstrated a 44% lower risk of death compared to those who started on placebo; although these results are controversion.8 The FDA AdComm panel voted that Albrioza’s data included in its application did not establish effective treatment for patients with ALS with a 6–4 vote (6 no; 4 yes). Several members cited concerns over the trial’s conduct and robustness of the data. Additionally, some felt as though the ALSFRS-R scores for patients in the placebo arm were high, which may have altered the perception of the data.9  On July 5, the FDA announced that its  Peripheral and Central Nervous System Drugs Advisory Committee is planning to reconvene on September 7, 2022, to discuss Albrioza’s application further. The Institute for Clinical and Economic Review (ICER) estimates Albrioza, will have an annual wholesale acquisition cost (WAC) of $169,000. ICER determined that at such a price, the drug would not be cost effective.10 ICER also noted in its draft report that policymakers can examine short-term pricing options, such as a price close to the production cost, until the benefits of the drug can be sufficiently assessed. The FDA-approved drugs for ALS include: riluzole which extends survival and/or time to tracheostomy and Mitsubishi Tanabe Pharma’s Radicava® (edavarone) for IV use/Radicava ORS (edavarone oral suspension) has been shown to slow the loss of physical function.

9/30/2022: futibatinib
The FDA is reviewing Taiho Oncology’s futibatinib for treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring FGFR2 gene rearrangements, including gene fusions. Futibatinib is an oral selective, and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3 and 4. The application was based on findings from the Phase 2b FOENIX-CCA2 trial which demonstrated that futibatinib produced an objective response rate (ORR) of 41.7% per independent central review with a median duration of response (DOR) of 9.7 months.11 Among patients who responded to treatment, 72% continued to respond for 6 months or longer. Similar products include Innovent Biologics’ Pemazyre® (pemigatinib) and QED Therapeutics’ Truseltiq® (infigratinib).



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