Specialty Pipeline Monthly Update: October 2022

This monthly pipeline wrap-up provides a review of newly approved specialty drugs, recent specialty drug launches, new indications and news of note on specialty drugs in the approval process. See separate article for pipeline information on traditional drugs.

New Drug Information

• Pedmark^® (sodium thiosulfate injection): The U.S. Food and Drug Administration (FDA) has approved Fennec’s Pedmark to reduce the risk of ototoxicity associated with cisplatin in pediatric patients one month of age and older with localized, non-metastatic solid tumors. Permanent hearing loss can be seen in approximately 60% of children treated with cisplatin and can be as high as 90%. Until now, interventions with management strategies such as cochlear implants and hearing aids only occurred after hearing loss had been detected and these interventions do not return normal hearing. Pedmark’s approval is based on two Phase 3 open label trials, SIOPEL 6 and COG ACCL0431, which demonstrated the incidence of hearing loss was significantly lower with Pedmark plus cisplatin arm compared with the cisplatin alone arm [21.4% vs. 73.3% in COG ACCLO431 and 32.7% vs. 63% SIOPEL6, respectively].¹Pedmark has launched with a wholesale acquisition cost (WAC) is $11,417.09 per vial.²
• Vegzelma^® (bevacizumab-adcd): The FDA has approved Celltrion’s Vegzelma for the treatment of six types of cancer:
  • metastatic colorectal cancer
  • recurrent or metastatic non-squamous non-small cell lung cancer (nsNSCLC)
  • recurrent glioblastoma
  • metastatic renal cell carcinoma
  • persistent, recurrent, or metastatic cervical cancer and
  • epithelial ovarian, fallopian tube, or primary peritoneal cancer.
• Vegzelma is a biosimilar of Genentech’s Avastin^® (bevacizumab). Currently, there are three other FDA approved Avastin biosimilars available. Vegzelma demonstrated in clinical trials that it is highly similar to the reference product in terms of efficacy, safety and pharmacokinetics.³ Vegzelma launch and price are pending.  
• Relyvrio^® (sodium phenylbutyrate/taurursodiol): Amylyx Pharmaceuticals’ Relyvrio has been granted accelerated approval by the FDA for the treatment of adults with amyotrophic lateral sclerosis (ALS). Relyvrio was approved based on Phase 2/3 trial that demonstrated changes on ALS Functional Rating Scale-Revised (ALSFRS-R) as the primary outcome. Findings demonstrated that Relyvrio treated patients had an ALSFRS-R score of 2.32 points higher than those on placebo.⁴ The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee did an about-face, voting 7-2 (7 yes; 2 no) in favor of recommending the therapy for FDA approval after previously voting against it back in March 2022, citing doubts in the drug’s efficacy. The $158,000 a year wholesale price of Relyvrio falls just below the annual cost of Radicava^® (edaravone), a Mitsubishi Tanabe Pharma ALS drug that was approved by the FDA in 2017. But Relyvrio’s price is still far higher than the $9,100 to $30,700 price range in which treatment with the drug would be cost effective, according to calculations by the Institute for Clinical and Economic Review (ICER).⁴
• Lytgobi^® (futibatinib): Taiho Oncology’s Lytgobi has been granted accelerated approval by the FDA for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma (iCCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements. Lytogobi was approved based on results of the primary analysis of the FOENIX*-CCA2 trial, a Phase 2 open-label trial that demonstrated an objective response rate (ORR) of 42% as measured by independent central review. The median duration of response (DOR) was 9.7 months, with 72% of responses lasting at least six months.⁵ Lytgobi’s launch and price are pending.
• Furoscix^® (furosemide injection): The FDA approved ScPharmaceuticals’ Furoscix, a formulation of furosemide delivered via an on-body infusor for the treatment of congestion due to fluid overload in adults with New York Heart Association Class II/III chronic heart failure. Furoscix is not indicated for emergency situations or in patients with acute pulmonary edema. Furoscix will deliver only an 80-mg dose. Furoscix is the first FDA-approved subcutaneous loop diuretic that delivers IV equivalent diuresis at home via the Furoscix infusor. Furoscix was approved based on its ability to demonstrate IV equivalence in which Furoscix demonstrated 99.6% bioavailability and eight-hour urine output of 2.7L which was similar to subjects receiving intravenous furosemide.⁶ ScPharmaceuticals has launched Furoscix with an average wholesale price (AWP) $986.40 per box.

New Indications

• Orkambi^® (lumacaftor and ivacaftor): The FDA expanded Vertex’s Orkambi pediatric indication for treatment of cystic fibrosis in patients aged 12 months to less than 24 months who are homozygous for the F508del mutation in the CFTR gene.
• Dupixent^® (dupilumab): Sanofi and Regeneron’s Dupixent has been approved by the FDA as the first drug indicated for treatment of adult patients with prurigo nodularis.

October News

• “RBX2660 now with the brand name Rebyota is a potential first-in-class microbiota-based live biotherapeutic that aims to reduce recurrent C. difficile infection. The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) issued a positive vote on Sept. 22, 2022, for Ferring Pharmaceuticals’ RBX2660 (now with the brand name Rebyota) to reduce recurrence of C. difficile infection (CDI) after antibiotic treatment. RBX2660 is microbiota-based live biotherapeutic that contains live microorganisms that are used as active substances. RBX2660 is a fecal microbiota transplantation therapy that was developed by Rebiotix, a Ferring company. The committee voted 13 to 4 indicating that the data were adequate to support the effectiveness of RBX2660 to reduce the recurrence of C. difficile infection in adults 18 years of age and older following antibiotic treatment. The committee also voted 12 to 4 with one abstention that the data were adequate to support the safety of RBX2660.7
• “Drug pricing watchdog ICER keeps its eye on what price point a drug is best suited for based on clinical benefit — and the group’s newest draft report takes a closer look at MS treatments compared to a new one awaiting the FDA’s final say. The watchdog focused on TG Therapeutics’ new, potential blockbuster ublituximab, which is pending FDA review, and compared it to other classes of disease-modifying therapies (DMTs, as ICER calls them) that are currently on the market. As for whether or not the drug would be cost effective if approved, the group gave ublituximab a placeholder price of $71,187 in wholesale acquisition cost, or $55,081 a year. That price is the same as Roche’s Ocrevus (ocrelizimab). Other drug prices ICER compared included natalizumab (Tysabri) at over $100,000 a year, Novartis’ Kesimpta (ofatumumab) at over $80,000 a year and dropping to $71,000 in years two and after, and then dimethyl fumarate, the generic form of Biogen’s Tecfidera, coming in at under $3,000 per year. “At its placeholder price, ublituximab did not meet typical thresholds for cost effectiveness when compared to the market-leading oral, in large part due to differences in net price,” the report added.”⁸
• “As per The Business Research Company’s “Cell And Gene Therapy Global Market Report 2022”, the cell and gene therapy market is expected to grow from $6.58 billion in 2021 to $8.57 billion in 2022 at a compound annual growth rate (CAGR) of 30.2%. The growth in the market is mainly due to the companies resuming their operations and adapting to the new normal while recovering from the COVID-19 impact, which had earlier led to restrictive containment measures involving social distancing, remote working, and the closure of commercial activities that resulted in operational challenges. The gene and cell therapy market is expected to reach $21.33 billion in 2026 at a CAGR of 25.6%. Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market.”⁹
• “Attempting to push forward an ALS drug despite a failed Phase 3 study, Biogen saw its FDA review date delayed. The big biotech announced that the agency pushed back its decision deadline by three months to April 25, 2023 for tofersen, an antisense drug designed to treat a genetic subset of ALS. Biogen had previously received priority review for the application, which will fall under the accelerated approval pathway. In a press release, Biogen said the FDA had requested additional information, but it’s not clear what they sought. A Biogen spokesperson told Endpoints News, “We aren’t able to provide further detail on our regulatory interactions, but information requests are a standard part of the FDA review process.” Biogen is trying to get tofersen approved for ALS patients with SOD1 genetic mutations, a population that makes up roughly 2% of the global patient population. Though there are many genes known to be implicated in ALS, SOD1 is the most common. Current research estimates that all genetic mutations account for about 10% of ALS cases worldwide.”¹⁰
• “Pegcetacoplan, an investigational complement factor inhibitor that has the potential to be the first FDA–approved treatment for geographic atrophy in dry age-related macular degeneration (AMD), reduced growth in geographic lesions in the retina after two years of treatment. The results, from two Phase 3 clinical trials, were presented at the American Academy of Ophthalmology(AAO) meeting. So there’s literally dozens of drugs being studied to treat this and the one that may be approved first — there’s no approved therapy now — but there is a drug called pegcetacoplan, which has a PDUFA date with the FDA of the end of November — November 26 — meaning that they’re going to report by November 26 if it’s FDA approved, which would be a huge milestone in this space as a potential treatment for geographic atrophy.”¹¹

References

1. https://www.globenewswire.com/news-release/2022/09/21/2520026/0/en/Fennec-Pharmaceuticals-Announces-FDA-Approval-of-PEDMARK-Sodium-Thiosulfate-Injection.html
2. https://fennecpharma.com/wp-content/uploads/2022/09/Colorado-Price-Disclosure-Letter.pdf
3. https://www.businesswire.com/news/home/20220927006146/en/Celltrion-USA-Receives-U.S.-FDA-Approval-for-its-Oncology-Biosimilar-Vegzelma%C2%AE-bevacizumab-adcd-for-the-Treatment-of-Six-Types-of-Cancer
4. https://medcitynews.com/2022/09/cost-of-amylyx-als-drug-revives-pricing-debate-for-new-meds-in-rare-diseases/
5. https://www.prnewswire.com/news-releases/fda-approves-taihos-lytgobi-futibatinib-tablets-for-previously-treated-unresectable-locally-advanced-or-metastatic-intrahepatic-cholangiocarcinoma-301638254.html
6. https://www.biospace.com/article/releases/scpharmaceuticals-announces-fda-approval-of-furoscix-furosemide-injection-the-first-and-only-self-administered-subcutaneous-loop-diuretic-for-the-at-home-treatment-of-congestion-in-chronic-heart-failure/
7. https://www.formularywatch.com/view/fda-advisory-committee-gives-positive-vote-for-microbiota-based-c-diff-therapy/
8. https://endpts.com/icer-on-ms-drugs-tgs-ublituximab-not-cost-effective-at-the-same-price-as-roches-ocrelizimab/
9. https://www.einnews.com/pr_news/596510292/cell-and-gene-therapy-global-market-estimated-to-grow-at-25-rate
10. https://endpts.com/fda-delays-biogens-als-drug-review-by-three-months-decision-to-now-come-next-april/
11. https://www.medpagetoday.com/meetingcoverage/aaovideopearls/101256?xid=nl_mpt_DHE_2022-10-17&eun=g1537885d0r&utm_source=Sailthru&utm_medium=email&utm_campaign=Daily%20Headlines%20Evening%202022-10-17&utm_term=NL_Daily_DHE_dual-gmail-definition/