Specialty Drug Pipeline Monthly Update: July 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

• Litfulo^® (ritlecitinib): The FDA has approved Pfizer’s Litfulo, a once-daily oral treatment, for individuals 12 years of age and older with severe alopecia areata. The approved recommended dose for Litfulo is 50mg. It is the first treatment approved by the FDA for adolescents (12+) with severe alopecia areata. Litfulo is a kinase inhibitor which inhibits Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases. Litfulo was approved based on the Phase 2b/3 trial, ALLEGRO which demonstrated 23% of patients treated with Litfulo 50mg had 80% or more scalp hair coverage (SALT≤20) after six months compared to 1.6% with placebo.¹ Litfulo will have a wholesale acquisition cost (WAC) of $49,000 a year. Lilly’s Olumiant^® (baricitinib), is FDA approved for adult patients with severe alopecia areata, has an annual WAC of about $31,500 to $63,000 depending on dose.
• Rystiggo^® (rozanolixizumab-noli): The FDA has approved UCB’s Rystiggo for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. Rystiggo a subcutaneous infusion administered by a healthcare professional, is a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG. Rystiggo was approved based on the Phase 3 MycarinG study which evaluated the primary efficacy endpoint of the change from baseline in the MG-ADL total score at day 43. MG-ADL is a measurement tool which assesses the impact of gMG on daily functions of eight signs or symptoms that are typically affected in gMG. Rystiggo demonstrated a statistically significant difference compared to placebo (-3.4 points in Rystiggo-treated group at either dose vs -0.8 points in the placebo-treated group).² Rystiggo will be available in the 3^rdquarter of 2023 with pricing to follow.
• Ngenla^® (somatrogon-ghla): The FDA has approved Pfizer’s Ngenla (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. Ngenla was approved based on the Phase 3 study which evaluated the safety and efficacy when administered once weekly compared to once-daily somatropin. The study met its primary endpoint of Ngenla non-inferiority compared to somatropin, as measured by annual height velocity at 12 months.³Ngenla is expected to become available in August 2023 with pricing to follow.
• Lantidra^® (donislecel-jujn): The FDA has approved CellTrans’ Lantidra, a cellular therapy for use in an uncommon, severe form of Type 1 diabetes in which patients are unable to approach A1C because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. It must be used in conjunction with concomitant immunosuppression. Lantidra consists of purified allogeneic pancreatic islets that are infused into the portal vein of the liver. One to three infusions may be administered depending on treatment response, but there are no data regarding the effectiveness or safety for patients receiving more than three infusions. Lantidra was approved based on two studies which totaled 30 participants. Twenty-five (83.3%) became insulin independent for any duration; four were insulin independent for less than one year, 11 for one to five years, and 10 for more than five years.⁴ Lantidra launch and price are pending.
• Roctavian™ (valoctocogene roxaparvovec-rvox): The FDA approved BioMarin Pharmaceuticals’ Roctavian gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. Roctavian was approved based on the Phase 3 GENEr8-1 study, in which 112 who received Roctavian experienced an average 52% reduction in their annualized bleeding rate (ABR) through three years of follow-up.⁵ BioMarin Pharmaceutical, assigned a WAC of $2.9 million for a one-time treatment.⁶
• Beyfortus™ (nirsevimab-alip): The FDA approved AstraZeneca’s Beyfortus for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) in newborns and infants born during or entering their first RSV season, and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. Beyfortus is a long-acting anti-RSV monoclonal antibody that is administered as a single dose timed to the RSV season. The approval by the FDA follows the unanimous vote by the Antimicrobial Drugs Advisory Committee (AMDAC) on the favorable benefit-risk profile of Beyfortus. Beyfortus was approved based on the Phase 2b trial, which demonstrated that treatment with Beyfortus significantly reduced the incidence of medically attended lower respiratory tract disease caused by RSV by 70.1% compared with placebo.⁷ Beyfortus will be available in the U.S. ahead of the upcoming 2023-2024 RSV season with pricing to follow.

New Indications

• Talzenna^® (capsule) (talazoparib tosylate): The FDA has expanded Pfizers’ Talzenna to include a new indication to be used in combination with enzalutamide, for the treatment of adults with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC).

July News

• “The Institute for Clinical and Economic Review (ICER) posted its revised evidence reportassessing the comparative clinical effectiveness and value of exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics) and lovotibeglogene autotemcel (“lovo-cel”, bluebird bio) for sickle cell disease…. Current evidence suggests that lovo-cel and exa-cel would achieve common thresholds for cost-effectiveness if priced between $1.35M to $2.05M.” ⁸
• “The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted favorably (10 yes, 4 no) on whether the available evidence supports the effectiveness of palovarotene as a treatment for fibrodysplasia ossificans progressiva (FOP).”⁹
• “The FDA’s Covid strain selectionfor the next round of shots puts Pfizer, Moderna and Novavax on track to deliver new jabs in time for the fall — a decisive win for the vaccine makers as they gear up to compete against one another. The FDA advised the three companies to manufacture single-strain jabs targeting the omicron subvariant 1.5, one of the most immune-evasive Covid variants to date. That strain accounted for nearly 40% of all Covid cases in the U.S. in early June, but that proportion is slowly declining, according to data from the Centers for Disease Control and Prevention.”¹⁰
• “Madrigal Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company pursuing novel therapeutics for nonalcoholic steatohepatitis (NASH), announced the completion of the rolling submission of its New Drug Application (NDA) to the FDA for resmetirom for the treatment of adults with NASH with liver fibrosis, a disease with no approved therapy. Madrigal has requested a priority review of the resmetirom NDA.”¹¹

References

1. https://www.fiercepharma.com/pharma/pfizers-litfulo-enters-scene-alopecia-adolescent-nod-rival-lillys-olumiant
2. https://www.prnewswire.com/news-releases/ucb-announces-us-fda-approval-of-rystiggo-rozanolixizumab-noli-for-the-treatment-of-adults-with-generalized-myasthenia-gravis-301864023.html
3. https://www.pfizer.com/news/press-release/press-release-detail/fda-approves-pfizers-ngenlatm-long-acting-once-weekly
4. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes
5. https://www.prnewswire.com/news-releases/us-food-and-drug-administration-approves-biomarins-roctavian-valoctocogene-roxaparvovec-rvox-the-first-and-only-gene-therapy-for-adults-with-severe-hemophilia-a-301867403.html
6. https://www.managedhealthcareexecutive.com/view/fda-approves-roctavian-the-first-gene-therapy-for-hemophiliaa
7. https://www.globenewswire.com/news-release/2023/07/17/2705911/0/en/Press-Release-FDA-approves-Beyfortus-nirsevimab-alip-to-protect-infants-against-RSV-disease.html
8. https://icer.org/news-insights/press-releases/icer-publishes-evidence-report-on-gene-therapies-for-sickle-cell-disease/
9. https://www.empr.com/home/news/drugs-in-the-pipeline/fda-panel-votes-on-palovarotene-for-fibrodysplasia-ossificans-progressiva/
10. https://www.cnbc.com/2023/06/20/covid-vaccines-pfizer-moderna-and-novavax-gear-up-for-fall-rollout.html
11. https://www.biospace.com/article/releases/madrigal-pharmaceuticals-completes-submission-of-new-drug-application-seeking-accelerated-approval-of-resmetirom-for-the-treatment-of-nash-with-liver-fibrosis/