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
- Imjudo®(tremelimumab-actl): The U.S. Food and Drug Administration (FDA) approved AstraZeneca’s Imjudo in combination with AstraZeneca’s Imfinzi® (durvalumab) for the treatment of adult patients with unresectable hepatocellular carcinoma (HCC), the most common type of liver cancer. The novel dose and schedule of the combination, which includes a single dose of the anti-CTLA-4 antibody Imjudo 300mg added to the anti-PD-L1 antibody Imfinzi 1500mg followed by Imfinzi every four weeks, is called the STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab). Imjudo was approved based on positive results from the HIMALAYA Phase 3 trial which demonstrated patients treated with the combination of Imjudo and Imfinzi experienced a 22% reduction in the risk of death compared to patients treated with sorafenib.1
- AstraZeneca/MedImmune’s Imjudo was also approved by the FDA in combination with AstraZeneca’s Imfinzi®(durvalumab) plus platinum-based chemotherapy for the treatment of adult patients with Stage IV (metastatic) non-small cell lung cancer (NSCLC). Imjudo was approved for this indication based on the Phase 3, POSEIDON trial, which demonstrated that Imfinzi and Imjudo combined with chemo and maintenance therapy achieved a median overall survival of 14 months compared to 11.7 months in patients on chemotherapy alone followed by maintenance therapy. Additionally, it had a median progression-free survival of 6.2 months and 4.8 months, respectively.2 Imjudo has launched with a wholesale acquisition cost (WAC) of $3,250 per 1.25mL vial.
- Tecvayli® (teclistamab-cqyv): The FDA has approved Janssen Pharmaceutical’s Tecvayli for the treatment of adult patients with relapsed or refractory multiple myeloma, who previously received four or more prior lines of therapy, including a proteasome inhibitor, immunomodulatory drug and anti-CD38 monoclonal antibody. Tecvayli is a first-in-class, bispecific T-cell engager antibody that is administered subcutaneously by a health care professional. The Phase 2 MajesTEC-1 clinical trial demonstrated an overall response rate (ORR) of 61.8 percent in patients treated with Tecvayli, with 28.2 percent of patients achieving a complete response (CR) or better.3The median time to first response was 1.2 months with a median follow-up of 7.4 months. The estimated duration of response (DOR) rate was 90.6 percent at six months and 66.5 percent at nine months.3 Tecvayli has launched with WAC of $1770 per 3mL vial.
- Elahere® (mirvetuximab soravtansine-gynx): The FDA has granted accelerated approval for ImmunoGen’s Elahere for the treatment of adult patients with folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Elahere was approved under FDA’s accelerated approval program based on the SORAYA trial which achieved a 32% ORR, with five patients completely cleared of tumors. The ORR was a decided improvement on the 12% figure seen in an earlier trial of single-agent chemotherapy.4 Continued approval may be contingent upon verification and description of clinical benefit in a confirmatory trial. Elahere is a first-in-class antibody-drug conjugates (ADCs) directed against FRα, a cell-surface protein highly expressed in ovarian cancer and is the first FDA approved ADC for platinum-resistant disease. Elahere can be used regardless of prior treatment with Roche’s Avastin. The broad label opens the treatment up to approximately 5,200 patients in the U.S.5 Elahere is supplied as a single-dose vial containing 100mg/20mL solution of mirvetuximab soravtansine-gynx. Treatment is administered as an intravenous infusion following dilution. Elahere will launch shortly with a 20mg dose of Elahere priced at $6,220. Patients typically receive three to four vials in a cycle of treatment, leaving the cost between approximately $18,500 and $25,000.
- Oxlumo® (lumasiran): The FDA expanded Alnylam’s Oxlumo indication to include the treatment of primary hyperoxaluria type 1 (PH1) to include reduction of plasma oxalate in patients with advanced PH1, including patients with end-stage kidney disease on dialysis
- Rinvoq® (upadacitinib): AbbVie’s Rinvoq has been granted a new indication by the FDA to include the treatment of adults with active non-radiographic axial spondyloarthritis (nr-axSpA) with objective signs of inflammation who have responded inadequately to NSAIDs.
- Cotellic® (cobimetinib): The FDA expanded Genentech (Roche)’s Cotellic indication to include treatment of adults with histiocytic neoplasm.
- Libtayo® IV (cemiplimab-rwlc): The FDA expanded Regeneron/Sanofi’s Libtayo IV indication to include the use in combination with platinum-based chemotherapy for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with no EGFR, ALK or ROS1 aberrations. Patients must either have metastatic or locally advanced tumors that are not candidates for surgical resection or definitive chemoradiation. Patients may be treated with this combination irrespective of PD-L1 expression or histology.
- “Drug pricing watchdog ICER released its final report on drug price increases in California that were unsupported by new clinical evidence, pointing to three outliers — Bristol Myers Squibb’s cancer drugs Revlimid and Sprycel, and Eli Lilly’s migraine drug Emgality. The report builds on a price transparency law passed in California in 2017, which requires manufacturers to report year-over-year spending increases to prescription drugs’ WAC prices, but does not include net prices, which ICER took issue with. The nonprofit watchdog said it winnowed down lists of the most expensive drugs in California from 2020, seeking input as to whether its figures on net prices, if available, were correct. While conversations with manufacturers on net prices canceled out many of the top ones listed, ICER said in all three of these cases, the price increases were high and unjustified by new clinical evidence.”6
- “Following two FDA rejections of novel oral anemia drugs, all eyes are on GSK’s daprodustat. Now, the drug has come out of an FDA panel meeting partially bruised, losing experts’ support in one of the two patient populations that it’s seeking approval for. After heated discussions during a meeting, an independent FDA advisory committee voted 11-5 that the benefit of GSK’s daprodustat doesn’t outweigh its risk in patients with chronic kidney disease-related anemia who are not on dialysis, but, in a win for GSK, the experts voted 13-3 in support of daprodustat’s proposed use in the dialysis-dependent population. The FDA doesn’t have to follow its advisory committee’s recommendation. If the votes eventually play out according to the FDA’s decisions, daprodustat could at least partially redeem setbacks previously suffered by two other agents in the novel HIF-PHI oral drug class. To most experts on the FDA advisory committee, daprodustat’s risks are simply too pronounced to be offset by the administration convenience it offers. The safety signals in the nondialysis-dependent group were more than just the risks of heart failure and bleeding gastric erosions, which were present in the dialysis group. For nondialysis patients, other risks included heart attack and stroke, among other potential complications. While the experts said they wanted to give patients not on dialysis another treatment option, many were just not satisfied by the data and advised that longer-term studies be conducted with the patient group instead.”7
- “More than two years ago, BioMarin went into a version of corporate shock when the FDA demanded to see two years of follow-up data from its Phase III trial ahead of a marketing decision for its hemophilia A gene therapy valoctocogene roxaparvovec (valrox). Execs put out word that the FDA has upped the ante one more time — putting a damper on any lingering hopes for a Q1 approval by the PDUFA date next year. In a release that went out the biotech — which has made much of its plans to launch the treatment for millions of dollars at each turn, with the support of ICER analysts — announced the regulatory changeup, saying that the agency did not tell the team of any specific change to the PDUFA date of March 31, 2023. But: “the Agency stated that submission of these results may qualify as a Major Amendment, which would extend the action date by 3 months. FDA will evaluate the additional data prior to making this determination.””8
- “While the FDA has only signed off on three interchangeable biosimilars so far — Viatris’ insulin Semglee, Boehringer Ingelheim’s Humira interchangeable Cyltezo (launching next year), and Coherus’ Lucentis interchangeable Cimerli — Jacqueline Corrigan-Curay, CDER’s principal deputy center director, explained to the Association of Accessible Medicine’s industry conference yesterday that the agency is willing to work with industry where the science is justified. She noted that in the case of Semglee, a clinical immunogenicity study comparing its insulin glargine product with Sanofi’s reference product Lantus was not considered necessary before earning the interchangeable tag. Likewise, Cimerli won approval as an interchangeable biosimilar without first running switching studies.”9