Specialty Pipeline Update: February 2022

New drugs, new indications and news of note

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

• Kimmtrak^® (tebentafusp-tebn): The United States Food and Drug Administration (FDA) approved Immunocore’s Kimmtrak^® (tebentafusp-tebn) for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma (mUM). The approval of Kimmtrak is based on the results of the Phase 3 IMCgp100-202 clinical trial in which Kimmtrak demonstrated a statistically and clinically meaningful overall survival (OS) benefit, hazard ratio of 0.51, with median OS of almost 22 months.¹ Kimmtrak has launched with an average wholesale price (AWP) of $22,512 per vial.
• Vabysmo^® (faricimab-svoa): The FDA approved Genentech’s Vabysmo (faricimab-svoa) for the treatment of wet, or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME). AMD and DME are two of the leading causes of vision loss. After four initial monthly doses, Vabysmo is delivered as injections from one to four months apart in the first year; the current standard of care for wet AMD and DME requires injections every one to two months. The FDA approved Vabysmo on the results from four Phase 3 studies: TENAYA and LUCERNE for wet AMD and YOSEMITE and RHINE for DME. All the trials demonstrated Vabysmo was noninferior to aflibercept and the incidence of ocular adverse events was comparable.² Vabysmo has launched with an AWP of $2,628 per vial.
• Enjaymo^® (sutimlimab-jome): Sanofi’s Enjaymo (sutimlimab-jome) was approved by the FDA to decrease the need for red blood cell transfusion due to hemolysis in adults with cold agglutinin disease (CAD). Enjaymo is the first approved treatment for people with CAD and works by inhibiting the destruction of red blood cells (hemolysis). As red blood cells have the vital job of carrying oxygen throughout the body, patients with CAD may experience severe anemia, which can result in fatigue, weakness, shortness of breath, light-headedness, chest pain, irregular heartbeat, and other potential complications. CAD impacts the lives of an estimated 5,000 people in the United States. Enjaymo was approved based on its clinical trial that met its primary efficacy endpoint, which was a composite endpoint defined as the proportion of patients who achieved normalization of hemoglobin (Hgb) level ≥12 g/dL or demonstrated an increase from baseline in Hgb level ≥2 g/dL at the treatment assessment time point (mean value from weeks 23, 25, and 26) and no blood transfusion from weeks 5 through 26 or medications prohibited per the protocol from weeks 5 through 26.³ Fifty-four percent of patients met the composite primary endpoint criteria with 63% of patients achieving a hemoglobin ≥ 12 g/dL or an increase of at least 2 g/dL; 71% of patients remaining transfusion-free after week five; and 92% of patients did not use other CAD-related treatments. Enjaymo is administered intravenously. Patients >75kg require seven vials of Enjaymo every two weeks in month one, followed by seven vials every other week.  Enjaymo has launched with an AWP of $2,160 per vial

New indications

• None

February news

• “Eli Lilly’s tirzepatide — the type II diabetes candidate and Trulicity’s heir apparent, projected to be a megablockbuster drug within the next five years — has been waiting on the FDA to give it the okay. Yet according to cost watchdog ICER, there is still some doubt about just how useful it can be to patients when compared to top rivals. The Institute for Clinical and Economic Review released its report on the drug candidate earlier today — giving evidence for the drug a “B+” rating and saying “the evidence provides high certainty that tirzepatide delivers at least a small net health benefit when added to background therapy, with the possibility of a substantial net health benefit.” When ICER took a vote on clinical effectiveness, here’s what ended up happening: A unanimous majority (13-0) found the evidence for tirzepatide is enough to demonstrate a net health benefit when tirzepatide added to background therapy is compared to background therapy alone. A slight majority (7-6) found that the evidence is not adequate to demonstrate a net health benefit when tirzepatide added to background therapy is compared to injectable semaglutide, the drug name for Novo Nordic’s Ozempic. A majority (10-2 with 1 abstention) found that the evidence is not adequate to demonstrate a net health benefit when tirzepatide added to background therapy is compared to empagliflozin, which is the drug name for Boehringer Ingelheim’s Jardiance.”⁴
• “The FDA said it’s no longer moving forward with its plan to authorize two doses (while waiting for data on a third dose) of Pfizer’s COVID-19 vaccine for the youngest group of children, from 6 months through 4 years of age. ‘Based on the agency’s preliminary assessment, and to allow more time to evaluate additional data, we believe additional information regarding the ongoing evaluation of a third dose should be considered as part of our decision-making for potential authorization,’ FDA’s top vaccine official Peter Marks and acting FDA commissioner Janet Woodcock said in a statement while announcing that next week’s VRBPAC meeting would be delayed.”⁵
• “When is a clinical trial conducted exclusively in a foreign country enough to support FDA approval? That was the question before the agency’s Oncologic Drugs Advisory Committee (ODAC) on Thursday as it weighed whether to recommend Eli Lilly and Innovent Biologics’ application for its PD-1 lung cancer drug Tyvyt (sintilimab). By a vote of 14-1, the independent panel ruled against the companies, declaring that another clinical trial, demonstrating that the drug works on U.S. patients, should be required before approval. The decision might have been different had Tyvyt been destined for a market with no good options for patients—or if the trial had used the gold standard of cancer research, overall survival, as its primary endpoint. But it didn’t, and Tyvyt was aiming to treat non-small cell lung cancer, a disease with multiple rival drugs that have proven they can extend patients’ lives.”⁶
• “It’s Eli Lilly’s year in 2022, with the top two biggest prospects belonging to the Big Pharma and taking nearly half that total with just shy of $11 billion in potential earnings from its prospective drugs in Alzheimer’s and diabetes, respectively. Overall, it’s the Alzheimer’s drug prospectsthat dominate the sales charts as a group, with Lilly’s donanemab worth potentially $6 billion by 2026 and Roche’s rival gantenerumab set to take $2.5 billion. That’s $8.5 billion in total potential in a disease area that hadn’t produced a new therapy for nearly two decades. But with Biogen’s ongoing woes with Aduhelm—which also had sky-high expectations last year but has faced what could be the worst launch in drug history—the Lilly and Roche predictions may be some of the riskiest we’ve seen in many years. It’s also a good launch year for Bristol Myers Squibb, which, like Lilly, has two drugs in the top 10. Its hopefuls: mavacamten in cardiomyopathy and deucravacitinib in psoriasis and autoimmune disorders are together worth $4 billion in predicted peak sales. But it’s not been an easy path to 2022 for all of the top 10: Johnson & Johnson and Legend Biotech were hit by an extended FDA review that pushed an expected decision from November to late February over manufacturing and technical concerns. And mavacamten had its own FDA delay to contend with. And, while Alzheimer’s drugs may be the riskiest proposition launch-wise, Reata Pharmaceuticals is at the highest risk of falling short of an approval. An FDA advisory committee unanimously rejected its kidney-disease-hopeful bardoxolone last year, putting its projected $2.2 billion in 2026 sales in serious jeopardy.”⁷

 References

1. https://ir.immunocore.com/news-releases/news-release-details/immunocore-announces-fda-approval-kimmtrakr-tebentafusp-tebn
2. https://www.ajmc.com/view/fda-approves-fariximab-to-treat-wet-amd-and-dme
3. https://www.globenewswire.com/news-release/2022/02/04/2379517/0/en/FDA-approves-Enjaymo-sutimlimab-jome-first-treatment-for-use-in-patients-with-cold-agglutinin-disease.html
4. https://endpts.com/benefits-of-lillys-diabetes-candidate-tirzepatide-arent-better-than-other-diabetes-drugs-says-watchdog-icer/
5. https://endpts.com/breaking-fda-postpones-next-weeks-adcomm-for-pfizers-covid-19-vaccine-for-the-youngest-age-group/
6. https://www.fiercepharma.com/pharma/lilly-and-innovent-s-china-trial-not-diverse-enough-to-apply-to-u-s-approval-for-lung-cancer
7. https://www.fiercepharma.com/special-report/10-most-anticipated-drug-launches-2022