Specialty Pipeline Monthly Update: January 2023

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

• Adstiladrin^® (nadofaragene firadenovec-vncg): The U.S. Food and Drug Administration (FDA) approved Ferring Pharmaceuticals’ Adstiladrin for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Adstiladrin is a non-replicating adenoviral vector-based gene therapy designed to deliver a copy of a gene encoding a human interferon-alfa 2b (IFNα2b) to the bladder urothelium. This medication is given directly into the bladder (called intravesicular), through a catheter, and left in the bladder for 1 hour and instilled once every three months. Adstiladrin was approved based on the open-label, single arm, Phase 3 CS-003 trial that demonstrated a complete response (CR) rate of 51% and a median duration of response of 9.7 months. Of those who achieved an initial CR, 46% continued to be free of high-grade recurrence at 12 months.¹Adstiladrin is scheduled to launch in the second half of 2023.
• Lunsumio^® (mosunetuzumab-axgb): Genentech’s Lunsumio has been awarded accelerated approval by the FDA for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. Lunsumio is administered as an intravenous infusion for a fixed-duration, which allows for time off therapy, and can be infused in an outpatient setting. Lunsumio was approved based on the Phase 2 study which demonstrated an objective response in 80% of patients with a majority maintaining responses for at least 18 months. Lunsumio demonstrated a complete response in 60% of patients. The median duration of response among those who responded was 22.8 months.² Lunsumio has launched with a wholesale acquisition cost (WAC) $594.06 per vial (1 mg/mL); $17,821.78 per vial (30 mg/30 mL).
• Briumvi^® (ublituximab-xiiy): TG Therapeutics’ Briumvi was approved by the FDA for the treatment of relapsing multiple sclerosis(RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. Briumvi is an anti-CD20 monoclonal antibody that can be administered in a one-hour infusion following the starting dose. The administration schedule of Briumvi consists of a day one infusion of 150mg administered in four hours, a day 15 infusion of 450mg administered in one hour, followed by 450mg infusions every 24 weeks administered in one hour. The ULTIMATE I & II Phase 3 trials, demonstrated Briumvi’s superiority over teriflunomide in significantly reducing the annualized relapse rate (ARR) (0.076 vs 0.188 in ULTIMATE I) (0.091 vs 0.178 in ULTIMATE II).³ Briumvi has launched with a WAC $9,833 per vial ($4,916.50 per month for maintenance).
• NexoBrid^® (anacaulase-bcdb): The FDA has approved MediWound’s NexoBrid for eschar removal in adults with deep partial thickness and/or full thickness thermal burns. NexoBrid is a topically administered, bromelain-based biological product containing a sterile mixture of proteolytic enzymes. The product selectively removes burn eschar within 4 hours without harming surrounding viable tissue. NexoBrid lyophilized powder and gel vehicle must be mixed prior to administration and should only be administered by a health care provider. NexoBrid was approved based on the Phase 3 DETECT which evaluated patients with deep partial and full thickness thermal burns up to 30% of total body surface area (BSA). The DETECT trial demonstrated 93% of eschar removal with NexoBrid at the end of the treatment compared to 4% from the vehicle group. NexoBrid was also associated with a lower incidence of surgical eschar removal 4% vs 72% for standard of care (SOC). The median time to eschar removal was reported to be one day with NexoBrid and 3.8 days with SOC. The estimated median time to 95% or greater wound closure was 31 days for the NexoBrid arm and 36 days for the SOC arm.⁴ NexoBrid was also studied in patients with deep partial and full thickness thermal burns of 5% to 24% BSA. The incidence of surgical eschar removal for the NexoBrid group was 22% compared with 77% for the SOC group. The estimated median time to 95% or greater wound closure was 33 days for the NexoBrid arm and 24 days for the SOC arm.⁴ NexoBrid is scheduled to launch second quarter of 2023 with pricing to follow.
• Olpruva^® (sodium phenylbutyrate): Acer Therapeutics’ Olpruva was approved by the FDA as an oral suspension, as adjunctive therapy to standard of care for patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase, ornithine transcarbamylase, or argininosuccinic acid synthetase. Olpruva was approved via the 505b2 pathway using Horizon Therap’s Buphenyl^® (sodium phenylbutyrate) powder.⁵ Olpruva launch and price are pending.
• Leqembi^® (lecanemab-irmb): The FDA approved Eisai’s Leqembi under the accelerated approval pathway for the treatment of Alzheimer’s disease (AD). Lecanemab is an anti-amyloid beta protofibril antibody. The approval is based on the Phase 2 data that demonstrated that Leqembi reduced the accumulation of Aβ plaque in the brain, but was unable to meet its primary endpoint of change from baseline on a weighted composite score consisting of selected items from three cognitive function scales. Eisai has submitted a supplemental biologics license application (sBLA) to the FDA for full approval under the traditional pathway referencing the Phase 3 confirmatory CLARITY AD trial which demonstrated that Leqembi treatment met the primary endpoint and reduced clinical decline on the global cognitive and functional scale, CDR-SB, compared with placebo at 18 months by 27%, a treatment difference in score change of -0.45 on the 18-point CDR-SB score. Eisai has announced that Leqembi will have an approximate WAC of $26,500 per year for a patient who weighs 74kg (163 pounds).⁶

New Indications

• Actemra^® (tocilizumab): The FDA expanded the indication of Genentech (Roche)’s Actemra for IV treatment of COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen, mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).
• Tymlos^® (abaloparatide): Radius Health’s Tymlos has been granted an additional indication to include the treatment of men with osteoporosis at high risk for fracture.

January News

• “Drug pricing watchdog ICER said that the uncertainty in the health benefits of two potential Alzheimer’s drugs makes it challenging to assess their future cost-effectiveness, according to a draft report. The report evaluates Biogen and Eisai’s lecanemab and Eli Lilly’s donanemab, both of which are amyloid-clearing antibody drugs due for FDA decisions in the coming weeks and months. The FDA’s deadline for a decision on lecanemab’s accelerated approval is Jan. 6, while the deadline for donanemab is estimated to be in February. For lecanemab to be cost-effective, ICER said it would have to be priced between $9,000 to $21,000 per year, assuming optimistic treatment benefits. And for donanemab to be cost-effective, it would have to be between $14,500 and $46,900. ICER noted on its donanemab calculations that the evidence of health benefit was insufficient, and that it assumed that donanemab would have the same effectiveness as lecanemab.”⁷
• “ICER re­it­er­at­ed that TG Ther­a­peu­tic­s’ ubli­tux­imab, a po­ten­tial new ther­a­py for mul­ti­ple scle­ro­sis is not cost ef­fec­tive at the price of $55,081 per year — the same price as Roche’s MS drug Ocre­vus. The fi­nal re­port was re­leased ex­act­ly one week be­fore the PDU­FA date for ubli­tux­imab. Ubli­tux­imab, for­mer­ly a part of the two-drug com­bo Ukoniq that was FDA-ap­proved for blood can­cer, was sub­se­quent­ly pulled off the mar­ket af­ter a Phase 3 read­out fa­vored the con­trol arm over the drug. It is now back be­fore FDA to treat RMS, or re­laps­ing forms of mul­ti­ple scle­ro­sis. In short, the re­view­ers con­firmed in its 115-page re­port that there is­n’t enough ev­i­dence to com­pare the ben­e­fit of ubli­tux­imab with oth­er mon­o­clon­al an­ti­bod­ies like Bio­gen’s na­tal­izum­ab, No­var­tis’ ofa­tu­mum­ab, and Roche’s ocre­lizum­ab and rit­ux­imab. How­ev­er, when com­pared to oth­er modal­i­ties like oral treat­ments, ICER said ubli­tux­imab looked to be at least com­pa­ra­ble or even bet­ter among fu­marates and oth­er ther­a­pies like fin­golimod, ozan­i­mod and pones­i­mod in terms of re­duc­ing an­nu­al­ized re­lapse rate and con­firmed dis­abil­i­ty pro­gres­sion. Af­firm­ing the draft re­port from the watch­dog group on ubli­tux­imab ear­li­er this year, ICER said, “The mod­eled mon­o­clon­al an­ti­body treat­ments, with­out rit­ux­imab, did not meet typ­i­cal thresh­olds for cost ef­fec­tive­ness when com­pared to the mar­ket-lead­ing oral, in large part due to dif­fer­ences in net price.”⁸

References

1. https://www.oncnursingnews.com/view/nadofaragene-firadenovec-gets-go-ahead-in-high-risk-non-muscle-invasive-bladder-cancer
2. https://www.cancerhealth.com/article/fda-approves-lunsumio-treatment-follicular-lymphoma#:~:text=The%20U.S.%20Food%20and%20Drug,type%20of%20non%2DHodgkin%20lymphoma
3. https://www.pharmacytimes.com/view/fda-approves-briumvi-for-relapsing-multiple-sclerosis
4. https://www.empr.com/home/news/severe-burn-therapy-nexobrid-gets-fda-approval/#:~:text=The%20Food%20and%20Drug%20Administration,sterile%20mixture%20of%20proteolytic%20enzymes.
5. https://www.empr.com/home/news/olpruva-approved-for-patients-with-urea-cycle-disorders/
6. https://investors.biogen.com/news-releases/news-release-details/fda-approves-leqembitm-lecanemab-irmb-under-accelerated-approval
7. https://endpts.com/icer-on-alzheimers-lecanemab-should-be-priced-lower-than-aduhelm/