GZ402665

Genzyme, a Sanofi Company · Phase 2 active Small molecule

GZ402665 is a BTK inhibitor Small molecule drug developed by Genzyme, a Sanofi Company. It is currently in Phase 2 development for Relapsed or refractory mantle cell lymphoma, Relapsed or refractory marginal zone lymphoma. Also known as: Olipudase alfa.

GZ402665 is a small molecule inhibitor of the Bruton's tyrosine kinase (BTK) enzyme.

GZ402665 is a small molecule inhibitor of the Bruton's tyrosine kinase (BTK) enzyme. Used for Relapsed or refractory mantle cell lymphoma, Relapsed or refractory marginal zone lymphoma.

Likelihood of approval

16.3% vs 15.3% industry baseline

If approved by FDA: likely 2031–2034

Steps remaining: Phase 3 → NDA/BLA submission

Confidence: Medium

Why this estimate

• Baseline phase 2 → approval rate +15.3pp
  Industry-wide phase 2 drugs reach approval ~15.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).
• Oncology Phase 2 attrition -2.0pp
  Oncology drugs have higher Phase 2-to-Phase 3 attrition than average — many fail to show OS benefit in larger studies.
• Big-pharma sponsor +3.0pp
  Genzyme, a Sanofi Company is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2031–2034	—
EMA	EU	2032–2035	+0.7 yr
MHRA	GB	2032–2035	+0.7 yr
Health Canada	CA	2032–2036	+0.9 yr
TGA	AU	2032–2036	+1.2 yr
PMDA	JP	2032–2036	+1.5 yr
NMPA	CN	2033–2037	+2.3 yr
MFDS	KR	2032–2036	+1.4 yr
CDSCO	IN	2032–2037	+1.8 yr
ANVISA	BR	2033–2037	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

At a glance

Generic name	GZ402665
Also known as	Olipudase alfa
Sponsor	Genzyme, a Sanofi Company
Drug class	BTK inhibitor
Target	BTK
Modality	Small molecule
Therapeutic area	Oncology
Phase	Phase 2

Mechanism of action

BTK is a key component of the B cell receptor signaling complex and plays a crucial role in the activation and proliferation of B cells. Inhibiting BTK has been shown to be effective in treating certain types of B cell malignancies.

Approved indications

• Relapsed or refractory mantle cell lymphoma
• Relapsed or refractory marginal zone lymphoma

Common side effects

• Neutropenia
• Thrombocytopenia
• Anemia

Key clinical trials

• A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France (PHASE2)
• Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
• Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (PHASE2, PHASE3)
• A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency (PHASE2)
• Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
• Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (PHASE1, PHASE2)
• Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients (PHASE1)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• GZ402665 CI brief — competitive landscape report
• GZ402665 updates RSS · CI watch RSS
• Genzyme, a Sanofi Company portfolio CI

Frequently asked questions about GZ402665

What is GZ402665?

GZ402665 is a BTK inhibitor drug developed by Genzyme, a Sanofi Company, indicated for Relapsed or refractory mantle cell lymphoma, Relapsed or refractory marginal zone lymphoma.

How does GZ402665 work?

GZ402665 is a small molecule inhibitor of the Bruton's tyrosine kinase (BTK) enzyme.

What is GZ402665 used for?

GZ402665 is indicated for Relapsed or refractory mantle cell lymphoma, Relapsed or refractory marginal zone lymphoma.

Who makes GZ402665?

GZ402665 is developed by Genzyme, a Sanofi Company (see full Genzyme, a Sanofi Company pipeline at /company/genzyme-a-sanofi-company).

Is GZ402665 also known as anything else?

GZ402665 is also known as Olipudase alfa.

What drug class is GZ402665 in?

GZ402665 belongs to the BTK inhibitor class. See all BTK inhibitor drugs at /class/btk-inhibitor.

What development phase is GZ402665 in?

GZ402665 is in Phase 2.

What are the side effects of GZ402665?

Common side effects of GZ402665 include Neutropenia, Thrombocytopenia, Anemia.

What does GZ402665 target?

GZ402665 targets BTK and is a BTK inhibitor.

Related

• Drug class: All BTK inhibitor drugs
• Target: All drugs targeting BTK
• Manufacturer: Genzyme, a Sanofi Company — full pipeline
• Therapeutic area: All drugs in Oncology
• Indication: Drugs for Relapsed or refractory mantle cell lymphoma
• Indication: Drugs for Relapsed or refractory marginal zone lymphoma
• Also known as: Olipudase alfa