J&J's Balversa wins quick blockbuster nod as first targeted therapy for bladder cancer

Targeted therapies that aim at specific genetic biomarkers have been widely adopted in several cancer types, but not in bladder cancer. But a new nod the FDA doled out Friday could change that—and potentially put developer Johnson & Johnson in line for $1 billion sales.

The FDA waved through Janssen’s Balversa (erdafitinib) for previously chemo-treated advanced or metastatic bladder cancer patients whose tumors have an FGFR3 or FGFR2 mutation, making it the first targeted therapy approved for the indication and the first FGFR inhibitor on the market.

J&J helmsman Alex Gorsky has previously dubbed the drug a potential blockbuster, and the company is pricing Balversa in the range of $10,080 to $22,680 for a 28-day supply, depending on the dosage, a Janssen spokesperson told FiercePharma, adding that the estimated average cost, $18,400, “is generally comparable to other oral oncology medicines.”

Mutations in the FGFR, or fibroblast growth factor receptor, account for about a fifth of all metastatic urothelial carcinomas, the most common type of bladder cancer, according to the FDA. A once-daily oral drug, Balversa works by inhibiting two forms of the FGFR alterations that can lead to increased tumor cell growth.

The drug nabbed accelerated approval from the FDA based on results from a small phase 2 clinical study. In the single-arm study of 87 bladder cancer patients who had undergone at least one prior chemotherapy treatment, Balversa elicited an overall response rate of 32.2%, including 2.3% complete responses, and it stalled tumor progression or death by a median of 5.4 months.

At last year’s American Society of Clinical Oncology annual meeting, data from the full BLC2001 study—which included 12 chemo-naïve patients—showed patients treated with Balversa lived a median of 13.8 months.

“Experts have been very encouraged by the data to date for erdafitinib,” Credit Suisse analyst Vamil Divan said in a short Friday note to clients. “Although it is a more targeted market opportunity, we believe it still represents an underappreciated JNJ pipeline asset.”

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Gorsky, for his part, on the company’s fourth-quarter earnings call in January named Balversa and recently approved antidepressant Spravato (esketamine) as two promising new drugs with more than $1 billion of peak revenue opportunity.

The company’s oncology franchise could use some fresh blood right now. Prostate cancer drug Zytiga, J&J’s best-selling cancer therapy, is facing cheap copycat competition after a failed patent tussle put $1.77 billion in 2018 full-year U.S. sales at danger.

Even though the full-year haul marked an impressive 44.2% jump over 2017, Q4 numbers could serve as an indication of what’s to come. After an appeals court denied J&J’s request for an injunction in November, several generics have entered the market, and U.S. Q4 sales of Zytiga immediately plummeted 12.7%, to $351 million.

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Besides Balversa, new additions to J&J’s oncology arsenal also include Erleada, which won an FDA green light last February in nonmetastatic prostate cancer. Barclays analyst Geoff Meacham at that time predicted Erleada could peak at $1.3 billion in sales. But Pfizer and Astellas’ blockbuster Xtandi has since joined the fray, with Bayer’s darolutamide waiting in the wings. And as Bernstein analyst Wimal Kapadia observed, the Bayer drug’s safety profile looks superior to its competitors and its efficacy in line with them.

Balversa itself could face some in-class competition such as Incyte’s FGFR 1/2/3 inhibitor pemigatinib, Basilea Pharmaceutica’s derazantinib and BridgeBio spinout QED Therapeutics’ infigratinib, which once belonged to Novartis.

To confirm Balversa’s clinical benefit, J&J is conducting a phase 3 trial dubbed Thor that pits the FGFR inhibitor against chemo and Merck & Co.’s PD-1 superstar Keytruda.