The Purpose of the Study is to Continue to Provide Pemigatinib to Patients With Advanced Malignancies.

1. Is the biology right?

• Somatic driver evidence: no · None of FGFR2, FGFR3, FGFR1, PDCD1 has somatic-mutation evidence in metastatic neoplasm

  Rule: Open Targets has somatic-mutation evidence for the target in this cancer. Source: Open Targets: FGFR2 in metastatic neoplasm, 2026-11-11
• Human genetic evidence: no · No germline or somatic evidence for FGFR2, FGFR3, FGFR1, PDCD1 in metastatic neoplasm

  Rule: Any germline or somatic human genetic evidence for the target in this cancer, counted apart from animal-model and literature evidence. Source: Open Targets: FGFR2 in metastatic neoplasm, 2026-11-11
• Pediatric target: yes · PDCD1 on the FDA list

  Rule: Target is on FDA's Pediatric Molecular Target List (relevant targets); membership alone neither requires nor waives a study. Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
• Haematologic cancer: no · Solid tumour

  Rule: From the indication, or the registry conditions when no indication matches. Source: ClinicalTrials.gov NCT04949191, 2026-11-11

  Published context: Likelihood of approval from phase 1: 7.5% haematologic vs 4.6% solid (BIO / Informa / QLS 2021, 2011–2020). Population-level, not a prediction.

• FGFR2 (via Pemigatinib): Evidence types in metastatic neoplasm: literature; literature listed separately from human genetics. Datasources: europepmc. · Open Targets: FGFR2 in metastatic neoplasm
• FGFR3 (via Pemigatinib): Evidence types in metastatic neoplasm: literature; literature listed separately from human genetics. Datasources: europepmc. · Open Targets: FGFR3 in metastatic neoplasm
• FGFR1 (via Pemigatinib): Evidence types in metastatic neoplasm: literature; literature listed separately from human genetics. Datasources: europepmc. · Open Targets: FGFR1 in metastatic neoplasm
• PDCD1 (via Retifanlimab): Evidence types in metastatic neoplasm: literature, clinical; literature listed separately from human genetics. Datasources: clinical_precedence, europepmc. · Open Targets: PDCD1 in metastatic neoplasm

• Not loaded on this app yet: Normal-tissue expression in vital organs. Source: Human Protein Atlas (ticket 36).
• Not loaded on this app yet: Pan-essential in cell lines. Source: DepMap (ticket 36).
• Not loaded on this app yet: Approved drug on this target. Source: Drugs@FDA (ticket 30).

2. Can the result be trusted?

• Randomised: no · Non-randomised

  Rule: Registry allocation field. Source: ClinicalTrials.gov NCT04949191, 2026-11-11
• Small denominator: yes · 10 enrolled; each patient moves a rate by 100 ÷ 10 = 10.0 points

  Rule: Enrolment under 30; the arithmetic is shown, not a benchmark. Source: ClinicalTrials.gov NCT04949191, 2026-11-11
• Primary endpoint is overall survival: no · Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

  Rule: A registered primary outcome measure names overall survival. Source: ClinicalTrials.gov NCT04949191, 2026-11-11

  Published context: Non-OS primary endpoint: odds ratio 2.79 for a positive phase 3 (Otsuka 2021, 400 phase 3 oncology trials, to 2020). Shown with the next figure: a surrogate is easier to hit, and often a weak guide to survival.

  Published context: 52% of surrogate-to-OS correlations are low (Prasad 2015, published to 2014). Shown with the figure above.
• Results overdue: no · Results posted 2024-09-19

  Rule: Primary completion (actual) more than 12 months ago and no results posted to the registry. Source: ClinicalTrials.gov NCT04949191, 2026-11-11

• Not loaded on this app yet: Treated and evaluable counts, assessor, confirmed response, follow-up and data cutoff. Source: Readout extraction (ticket 32).
• Not loaded on this app yet: Primary endpoint changed between registry versions. Source: Registry versions in the store (ticket 32).

3. What must it beat?

• Not loaded on this app yet: Control-arm result and approved therapies for the same segment, with label links. Source: Drugs@FDA, FDA labels and readouts (tickets 30, 32).

4. Too crowded, or too late?

• Stopped on this target: 127 terminated, withdrawn or suspended trials of drugs against FGFR2; 21 give no reason. Reasons are quoted as the registry gives them on the target page. · FGFR2: what already stopped

• Not loaded on this app yet: Programs on the target by phase and months behind the leader. Source: Programs and Drugs@FDA (ticket 33).

5. Can it be approved, and stay approved?

• Single-country: no · 10 sites in 4 countries; 5 in the United States (50%)

  Rule: All listed sites are in one country; the share of US sites is shown. Source: ClinicalTrials.gov NCT04949191, 2026-11-11
• EU paediatric investigation plan: yes · EMEA-003104-PIP02-22 (Favezelimab;pembrolizumab, Treatment of Hodgkin lymphoma): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), date not given; EMEA-003063-PIP03-22 (vibostolimab;pembrolizumab, Treatment of melanoma): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), 27 Oct 2023; EMEA-003063-PIP02-22 (vibostolimab;pembrolizumab, Treatment of Hodgkin lymphoma): P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s), date not given; EMEA-002798-PIP04-23 (retifanlimab, Treatment of lung cancer): W: decision granting a waiver in all age groups for all conditions or indications, 29 Dec 2023; +11 more

  Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
• EU orphan designation: yes · EU/3/22/2743 positive 13 Jan 2023: Treatment of Merkel cell carcinoma; EU/3/18/2066 positive 24 Aug 2018: Treatment of biliary tract cancer; EU/3/20/2343 positive 19 Oct 2020: Treatment of anal cancer; EU/3/19/2216 positive 17 Oct 2019: Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK

  Rule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
• Authorised in the EU: yes · Pemazyre: authorised since 26 Mar 2021, orphan medicine, conditional; Keytruda: authorised since 17 Jul 2015; Zynyz: authorised since 19 Apr 2024, orphan medicine; Zynyz: application withdrawn

  Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
• FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the link

  Rule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
• FDA pediatric oncology approval: yes · Pembrolizumab and berahyaluronidase alfa-pmph [Keytruda Qlex], 19 Sept 2025: Treatment of adult and pediatric patients 12 years and older with unresectable or metastatic tumor mutational burden high (TMB-H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options; Pembrolizumab and berahyaluronidase alfa-pmph [Keytruda Qlex], 19 Sept 2025: Adjuvant treatment of adult and pediatric patients 12 years and older with Stage IIB, IIC, or III melanoma following complete resection; Pembrolizumab and berahyaluronidase alfa-pmph [Keytruda Qlex], 19 Sept 2025: Treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR) solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options; +7 more

  Rule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
• Registered studies open to children: yes · 76 interventional studies of Pemigatinib, Retifanlimab, Pembrolizumab include patients under 18; 45 are recruiting, not yet recruiting or active

  Rule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-11

• Caveat: Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where the sites are is a fact about the record, not a judgement of its quality.
• Rare pediatric disease voucher: FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. · FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs

• Not loaded on this app yet: Precedent approvals for target, modality and endpoint; accelerated-approval status. Source: Drugs@FDA and FDA accelerated-approval tables (ticket 30).

6. Can the sponsor finish?

• Sponsor: Incyte Corporation (industry) · ClinicalTrials.gov NCT04949191
• study director: Peter Langmuir, Incyte Corporation · Person page

• Not loaded on this app yet: Filed cash, quarterly spend and the runway arithmetic. Source: SEC XBRL company facts (ticket 37).
• Not loaded on this app yet: Sponsor's completed vs stopped oncology trials and results-reporting record. Source: Registry, by company (ticket 37).

What public data cannot tell you

Public records cannot show: sales forecasts; a probability of success for this asset; full deal terms and net prices; FDA designations and action dates before approval (shown only as company-reported); clinical holds unless the company disclosed them; manufacturing; patents on unapproved drugs; whether preclinical findings have been replicated.