Specialty Pipeline Update: July 2022

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

• None

New Indications

• Xalkori^® (Crizotinib): The FDA expanded Pfizer’s Xalkori indication to include inflammatory myofibroblastic tumor (IMT).
• Kyprolis^® (carfilzomib): Onyx (Amgen)’s Kyprolis has been granted an additional indication by the FDA to be used in combination with Sarclisa (isatuximab-irfc) and dexamethasone (Isa-Kd) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received one to three lines of therapy.
• Breyanzi^® (lisocabtagene maraleucel, also known as liso-cel): The FDA expanded the indication of BMS’s Breyanzi to include the treatment of adults with relapsed or refractory large B-cell lymphoma (LBCL) after failure of first-line therapy.
• Tafinlar^® (dabrafenib) and Mekinist (trametinib): The FDA expanded the indication of Novartis’ Tafinlar and Mekinist to include adult and pediatric patients aged six years and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternatives.
• Imcivree^® (setmelanotide): Rhythm Pharmaceuticals’ Imcivree has been granted an additional indication by the FDA including treatment of obesity and control of hunger in adult and pediatric patients six years of age and older with Bardet-Biedl syndrome (BBS).

JULY News

• “BioMarin’s commercial aspirations for its hemophilia gene therapy remain on ice after the FDA lobbed additional questions for the company to address, pushing back an expected approval submission to September. BioMarin was vague on what the FDA is asking for, only specifying that the agency had requested additional details and analyses rather than more studies. The company also pointed out that updated three-year safety data of its Phase 3 GENEr8-1 study will become available during the likely review period, although it was unclear whether the FDA would be requesting this. The Tuesday updatewas tucked into the release of additional data of a phase 1/2 trial, which noted a 95% reduction in the average annualized bleed rate over six years for patients with hemophilia A who took the drug. The FDA’s request pushes back the likely regulatory submission to some point ‘by the end of September,’ according to BioMarin.”¹
• “Roche is hyping up the early financials coming in for its eye disease therapy Vabysmo as the Swiss major looks to take on the might of Regeneron and Bayer’s major blockbuster, Eylea. Despite only being approved at the start of the year and the launch coming several months later, Roche saw Q2 sales hit a healthy CHF 109 million ($112 million), which the pharma said in an accompanying investor presentation  ‘showed an excellent uptake.’ Roche said that more than 70,000 vials have been distributed in the first five months of its U.S. launch and that it was seeing ‘strong customer uptake, with switching coming primarily from aflibercept [Eylea].’” Back in January the drug nabbed a green light for wet age-related macular degeneration (AMD) as well as a label to treat diabetic macular edema (DME), which makes Vabysmo the lone injectable therapy approved simultaneously in the U.S. for both conditions. The pharma said it also reckons the global retina market in total is set to grow to $15 billion by 2025, led by AMD. Roche expects nearly $9 billion to come from the AMD market alone. The company also believes there will be “rapid market transition to next-generation products” such as Vabysmo. The Roche med is a bispecific antibody and is the first and only FDA-approved treatment designed to block both VEGF and Ang-2., while other approved therapies block VEFG.²
• “The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) discussion of elivaldogene autotemcel (eli-cel) for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor. On the question “Do the benefits of eli-cel outweigh the risks, for the treatment of any sub-population of children with early active cerebral adrenoleukodystrophy (CALD)?” the CTGTAC voted 15 (yes) to 0 (no). The CTGTAC also discussed the overall safety of lentiviral vector (LVV) gene therapies, concluding in a 13 to 1 vote that the safety data from lovo-cel for sickle cell disease is not relevant to the review of eli-cel. In addition to granting eli-cel BLA priority review, the FDA previously granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation. bluebird bio is eligible to receive a priority review voucher upon potential approval of eli-cel.”³
• “Cost-effectiveness watchdog ICER took a look at several different drugs approved for obesity and weight loss — and to them, there is a clear winner. ICER determined that Vivus’ Qsymia, the brand name for the appetite suppressant phentermine combined with the anticonvulsant topiramate, was more cost effective for weight loss than other competitors such as Novo Nordisk’s Saxenda and Wegovy.”⁴

References

1. https://www.fiercebiotech.com/biotech/biomarin-hemophilia-gene-therapy-further-delayed-amid-new-fda-questions
2. https://www.fiercepharma.com/marketing/roche-hails-excellent-uptake-eylea-rival-vabysmo-it-lauds-consumers-switching-its
3. https://investor.bluebirdbio.com/news-releases/news-release-details/fda-advisory-committee-unanimously-endorses-eli-cel-gene-therapy
4. https://endpts.com/vivus-qsymia-beats-out-weight-loss-competitors-in-pricing-watchdogs-latest-cost-effectiveness-analysis/