Specialty Portfolio Update: March 2023 - Prime Therapeutics LLC (2023)

prospects

This monthly project overview provides an overview of newly approved specialty drugs, recently launched specialty drugs, new indications, and important news about specialty drugs in the approval process. More information on piping can be found in separate articles.traditionaldrugs,BiosimilaresYgene/cellTherapy.

Information about new drugs

• Altuviiio^®[antihemophilic factor (recombinant), Fc-VWF-XTEN-ehtl fusion protein]:The United States The Food and Drug Administration (FDA) has approved Sanofi's Altuviiio, a first-in-class long-acting factor VIII replacement therapy. Altuviiio is indicated for routine prophylaxis and reliever treatment to control bleeding episodes, and for perioperative treatment (surgery) in adults and children with hemophilia A. Altuviiio is the first treatment for hemophilia A that produces hemophilia A activity levels factor VIII normal or near normal (greater than 40%) for most of the week when given once weekly and significantly reduces bleeding compared with prior factor VIII prophylaxis. The FDA approval was based on the Phase 3 XTEND-1 clinical trial, which showed that once-weekly Altuviiio prophylaxis met the primary endpoint of providing significant protection against bleeding for people with severe hemophilia A with a mean annual bleeding rate (ABR) of 0.70 and a mean achieved ABR of 0.0.¹Altuviiio is expected to be commercially available in April at a price equivalent to the annual cost of treating one prophylactic patient with Eloctate^®that's $12 per unit, or a cost of $12,000 for a patient requiring 1,000 IU per week, or $625,000 per year.²
• Skyclarys™ (Omaveloxolona):The FDA has approved Reata Pharmaceuticals' Skyclarys for the treatment of Friedreich's ataxia in adults and adolescents 16 years of age and older. Friedreich's ataxia is a rare, genetically determined neurodegenerative disease. Patients with Friedreich's ataxia usually present with symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue. About 5,000 patients in the United States are currently diagnosed with Friedreich's ataxia. Skyclarys was approved based on a clinical study showing that patients treated with Skyclarys had a statistically significantly lower score on the Modified Friedreich Ataxia Rating Scale (mFARS) at week 48 compared to placebo, representing a placebo-corrected difference between groups of -2.41 points.³Skyclarys is available in 50mg capsules. Skyclarys has entered the market with an average wholesale price (AWP) of $37,000 per bottle (90 capsules).
• Daybue^®(Trophiniden): The FDA approved Daybue from Acadia Pharmaceuticals for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue is the first drug approved by the FDA for Rett syndrome. Rett syndrome is caused by a genetic mutation in which patients develop normally during childhood but later deteriorate and lose speech, hand motor control, and other functions. There are approximately 4,500 patients diagnosed with Rett syndrome in the United States. Daybue is a twice-daily solution that reduces inflammation in the brain, prevents certain types of cells from becoming overactive, and increases levels of a natural protein called IGF-1. Daybue was approved based on a phase 3 lavender study that showed that treatment with Daybue improved patients' symptoms compared to placebo in two questionnaire rating systems.⁴Patients reported consistent benefits. Daybue is expected to be available in late April at an average list price of approximately $575,000 to $595,000 per year, or $21.10 per milliliter for weight-based dosing.

new indications

• Version^®(abemaciclib): The FDA has approved an expanded indication for Lilly's Verzenio in combination with endocrine therapy (ET) for the adjuvant treatment of adult patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2 (HER2)-negative mammary nodes. -). early stage cancer (EBC) with high risk of recurrence. High-risk patients eligible for Verzenio can now be identified solely on the basis of node status, tumor size, and tumor grade (4+ nodes positive or 1-3 nodes positive and at least one of the following: 5 cm in size or grade 3 ). This expanded adjunctive indication eliminates the Ki-67 score requirement for patient selection.
• Kevzara^®(sarilumab):The FDA has expanded the indication for Sanofi's Kevzara and Regeneron to include the treatment of adults with polymyalgia rheumatica (PMR) who have had an inadequate response to corticosteroids or who are intolerant of corticosteroid dose reductions.
• jelly^®(including limb-gxly): The FDA has granted GlaxoSmithKline (Tesaro) and Jemperli of AnaptysBio a transition from accelerated to full approval with a revised indication for the treatment of patients with advanced or recurrent endometrial cancer due to mismatch repair (dMMR) deficiency ), as determined by an FDA-approved test to have progressed on or after platinum-based therapies and are not candidates for curative surgery or radiation.

march news

• “A sudden change of heart at the FDA has caused Sarepta Therapeutics shares to plummet. Sarepta did so two weeks after announcing that the FDA would evaluate its gene therapy without an advisory committee meeting.revealedThe agency will now convene experts to review data on his presentation of Duchenne muscular dystrophy (DMD). Late last month, Sarepta informed investors that it had completed the mid-cycle review for the SRP-9001 submission, and while no significant clinical or safety issues emerged, the FDA had decided to proceed without an advisory committee meeting. . The decision triggered a jump in Sarepta's share price and put the company on track to potentially receive regulatory approval before May 29 without undergoing independent peer review."⁵
• “BioMarin Pharmaceutical Inc. announced that it has received notification from the FDA that the agency has expanded the review of the company's Biologics License Application (BLA) for the Roctavian™ (valoctocogene roxaparvovec) gene therapy for adults with hemophilia A. serious. The FDA has determined that the submission of the three-year data analysis from the ongoing Phase 3 GENEr8-1 study represented a significant change due to the significant amount of additional data and established a new PDUFA target action date of January 30. June 2023. The company previously advised that this data transfer could qualify as a material amendment.”⁶
• “Ahead of the advisory committee hearing to discuss Biogen's ALS drug Tofersen, the FDA expressed its willingness to approve the drug, recently released briefing papers show. Noting the need for flexibility in a devastating disease like ALS, regulators signaled their willingness to give Tofersen the green light despite the failure of a pivotal study due to its effects on a specific protein implicated in ALS. The documents come after the regulatory flexibility was part of the same rationale the agency expressed in approving an ALS drug from Amylyx Pharmaceuticals last September, signaling the FDA's openness to approving new treatments for the disease. "Despite the notable limitations of a failed study and the many post-hoc exploratory analyzes performed after [the Phase III study], the department believes that the data could indicate a treatment effect of Tofersen on SOD1-ALS," he wrote. the fda. “This is a very rare and devastating disease; It is therefore paramount that we fully consider all available data." Tofersen did not achieve the primary endpoint of his phase 3 VALUE study because he was unable to show that it was better at delaying decline in function in patients with ALS SOD1 than placebo The results, reported in October 2021, showed that the observable effect of the drug on function was most likely due to chance that Tofersen achieved the desired result.Biogen said the p-value for the primary endpoint was p = 0.97".⁷
• "The FDA is working to expedite gene therapy development, including promoting the use of biomarkers to achieve accelerated approval of therapies for serious diseases," an agency official said. Biomarkers such as heart rate and blood pressure are characteristics of the body that can be measured. The FDA will support the use of biomarkers as surrogates for other biological markers in gene therapy clinical trials to achieve "expedited approval," said the agency's Peter Marks.⁸

references

1. https://www.sanofi.com/es/sala-de-prensa/comunicados-de-prensa/2023/2023-02-23-21-00-00-2614759
2. https://www.sanofi.com/es/sala-de-prensa/comunicados-de-prensa/2023/2023-02-23-21-00-00-2614759
3. https://www.neurologylive.com/view/fda-aprueba-omaveloxolona-primer-tratamiento-friedreich-ataxie
4. https://www.rettsyndrome.org/trofinetideapproved/
5. https://www.fiercebiotech.com/biotech/fda-shocks-sarepta-ordering-adcomm-dmd-gene-therapy-sudden-change-heart
6. https://investors.biomarin.com/2023-03-06-BioMarin-offers-Update-on-FDA-Review-of-ROCTAVIAN-TM-Valoctocogene-Roxaparvovec-Gene-Therapy-for-Adults-with-Severe- Hemophilia-A
7. https://endpts.com/fda-indicates-willingness-to-approve-biogen-als-drug-pese-failed-phiiii-study/
8. https://www.reuters.com/world/us/us-fda-official-says-agency-needs-start-using-accelerated-approval-gene-2023-03-20/

About Prime Therapeutics

Prime Therapeutics LLC (Prime) is a diversified pharmaceutical solutions organization serving health care plans, employers and government programs. Prime is jointly owned by 19 Blue Cross and Blue Shield plans, subsidiaries or affiliates of those plans. Magellan Rx Management, a Prime Therapeutics LLC company, is a pioneer in specialty and drug management and a leader in serving public sector government programs. Together, Prime and Magellan Rx offer a broad range of solutions for clients that bridge the gap between medical and pharmacy management. For more information visitwww.primetherapeutics.comYwww.magellanrx.com, or follow us on Twitter at @Prime_PBM and @Magellan_Rx.

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