Product information and disease description Place of product in therapy Approaches to treatment

Other product development or post-marketing obligations required by the FDA

8 evidence topics · 2 sources

Postmarketing requirement 5057-1: randomized trial of 70 mg once daily compared with a lower dosage

“Conduct a randomized clinical trial to further characterize known serious risks with lirafugratinib including severe stomatitis, palmar-plantar erythrodysesthesia syndrome, retinal pigment epithelial detachment, and nail toxicities, and to compare the safety and activity of lirafugratinib 70 mg once daily versus a lower dosage in patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or other rearrangements, who have not received prior FGFR inhibitors.” (opens the source at this quote in a new tab)
“Finally, we have determined that only a clinical trial (rather than a nonclinical or observational study) will be sufficient to assess these serious risks.” (opens the source at this quote in a new tab)

Postmarketing requirement 5057-1: timetable

Milestone	Date
Draft protocol submission	“01/2027”
Final protocol submission	“05/2027”
Trial completion	“11/2031”
Final report submission	“05/2032”

Postmarketing requirement 5057-2: drug interaction trial with a sensitive P-gp and BCRP substrate

“Conduct a clinical pharmacokinetic trial to assess a serious potential risk of increased drug toxicity by evaluating the effect of repeat doses of lirafugratinib on the single dose pharmacokinetics of a sensitive P-gp and BCRP substrate and inform appropriate management strategies for clinically relevant drug interactions.” (opens the source at this quote in a new tab)

Postmarketing requirement 5057-2: timetable

Milestone	Date
Draft protocol submission	“01/2027”
Final protocol submission	“05/2027”
Trial completion	“05/2032”
Final report submission	“09/2032”

Postmarketing commitment 5057-3: companion diagnostic validation

“Conduct adequate analytical and clinical validation studies to support revisions to the labeling of lirafugratinib to specify the use of an FDA-authorized in vitro companion diagnostic device to accurately and reliably identify patients using blood and/or tissue samples who have FGFR2 fusions and other rearrangements and for whom lirafugratinib therapy is indicated.” (opens the source at this quote in a new tab)
“The final study report should be accompanied by a labeling supplement to incorporate information about the use of an FDA-authorized companion diagnostic device.” (opens the source at this quote in a new tab)

Postmarketing commitment 5057-3: timetable

Milestone	Date
Study completion	“01/2029”
Final report submission	“09/2029”

Pediatric Research Equity Act requirement

“We are waiving the pediatric studies requirement for this application, in pediatric patients less than 17 years of age because necessary studies are impossible or highly impracticable, due to the exceedingly low incidence of FGFR2 altered tumors.” (opens the source at this quote in a new tab)

Supplemental application planned for other FGFR2-altered solid tumors

“The FDA recommended that the company first file an NDA for FGFR2-driven CCA, followed by a supplemental NDA for FGFR2-altered other solid tumors with data from an expanded cohort of patients.” (opens the source at this quote in a new tab)