Givosiran Sodium Drug Asset Due Diligence Report 2026

This Givosiran Sodium Drug Asset Due Diligence Report was built with PatSnap Life Sciences MCP workflows. Drug & Asset MCP establishes identity, ownership and stage; Clinical Trials MCP checks design, endpoints and readouts; Company & Deal Intelligence MCP reconstructs transaction precedent. Explore the MCP servers used in this report.

Decision date: 15 July 2026. Currency fields are presented in US$ millions as returned by the deal dataset. This is a screening memorandum, not legal, medical, patent or investment advice.

Approved

Highest phase

Registered trials

Result records

Matched deals

Executive recommendation: GO

Decision memo

Advance diligence: development maturity and available evidence support continued investment, subject to indication-specific safety, IP, and commercial gates.

The central underwriting question is whether Givosiran Sodium can convert its siRNA profile and ALAS1 biology into clinically meaningful differentiation while preserving an investable safety, IP and commercial position.

1. Asset identity and development status

Asset	Givosiran Sodium (query alias: Givosiran Sodium)
Modality / target	siRNA; ALAS1; ALAS1 inhibitors, RNAi
Highest global status	Approved
Originator	Alnylam Pharmaceuticals, Inc.
Active developers	Alnylam Australia Pty Ltd., Alnylam Netherlands BV, Alnylam Japan KK

The MCP disease footprint includes Porphyria, Acute Hepatic. The highest-phase flag is a useful orientation point, but the licensing case depends on indication-level evidence and rights, not the global label alone.

2. Clinical program: design and endpoint audit

Registry	Phase	Status	Enrollment	Lead primary endpoint
NCT03338816	Phase 3	Completed	94	Annualized Rate of Porphyria Attacks in Participants With Acute Intermittent Porphyria (AIP)
NCT03505853	Phase 1	Completed	10	Profile of Pharmacokinetics (PK) of Cytochrome P450 (CYP) probe cocktail
JPRN-jRCT2071200074	Not Applicable	研究終了	10	- Urinary aminolevulinic acid (ALA) levels - Urinary porphobilinogen (PBG) levels - Rates of porphyria attacks and hemin administration - Patient-reported outcomes, assessed by the Givosiran Patient Experience Questionnaire (GPEQ) at end of study - Frequency of adverse events

The trial set should be diligenced for randomization, comparator relevance, endpoint hierarchy, analysis population, multiplicity, geographic mix and readout timing. For early-stage studies, safety and dose selection can be as decision-critical as response rate.

Reproduce the asset-to-trial workflow with PatSnap MCP

3. Clinical readouts: what is known

Efficacy and safety of givosiran in Japanese patients with acute hepatic porphyria: clinical findings from an expanded access study

Not Applicable; n=10; evaluation: Positive. Reported fields: GPEQ = showed symptomatic improvement in eight participants

A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1

Phase 1/2; n=16; evaluation: not stated. Reported fields: Percentage of Participants With Adverse Events (AEs) = 100 percentage of participants ; -; -

Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study

Phase 3; n=94; evaluation: Positive. Reported fields: annualized attack rate = 1.4 unit ; annualized attack rate = 1.0 unit

These fields are structured evidence signals, not a substitute for statistical review. The next diligence pass should reconcile denominators, confidence intervals, follow-up, censoring, dose cohorts and treatment-emergent toxicity against the original abstract, registry and protocol.

4. Market and competitive position

Givosiran Sodium addresses Porphyria, Acute Hepatic. Commercial attractiveness rests on addressable biomarker-positive patients, treatment-line placement, duration, administration burden, pricing and displacement of entrenched standards. The modality—siRNA—must demonstrate a benefit large enough to offset class-specific safety and operational costs.

The strongest market-validation signal in this screen is partner behavior: 4 matched transaction record(s) indicate that sophisticated counterparties have assigned strategic value to the asset or its rights. That does not establish net present value; probability of success, remaining R&D spend, royalties, cost sharing and territorial scope still need modeling.

5. Transaction precedent and economics

Date	Transaction	Phase at deal	Disclosed economics
2020-07-08	Alnylam Pharmaceuticals and Taiba Group Partner to Commercialize RNAi Therapeutics in the Gulf States	Approved	Financial terms not disclosed
2019-12-12	PANTHERx® Rare Pharmacy Selected by Alnylam Pharmaceuticals to Distribute GIVLAARI™ (givosiran)	Phase 3	Financial terms not disclosed
2019-08-13	Ironwood Pharmaceuticals and Alnylam Pharmaceuticals Enter U.S. GI Disease Education and Promotional Agreement for Alnylam’s Givosiran in Acute Hepatic Porphyria (AHP)	NDA/BLA	US$9.5M stated total

Headline values are not directly comparable. Diligence should normalize upfront cash, equity, development and sales milestones, tiered royalties, opt-in mechanics, cost sharing, change-of-control clauses and geography.

6. IP and freedom-to-operate screen

The milestone feed surfaced a patent-application signal described as “Compositions and methods for inhibiting expression of the alas1 gene”. The milestone feed surfaced a patent-application signal described as “Compositions and methods for inhibiting expression of the alas1 gene”.

The claim chart should separately test composition or sequence coverage, formulation and dosing, indication and biomarker claims, combinations, manufacturing know-how, prosecution history, term extensions and third-party blocking rights. Confirm that licensed patents, data and know-how track every granted territory and field.

7. Principal risks and diligence gates

Class crowding and differentiation versus other PD-(L)1/VEGF agents
Immune and anti-angiogenic safety, including bleeding and cardiovascular risk
Biomarker strategy, indication selection, and head-to-head endpoint execution

Cross-functional diligence should also test CMC comparability, supply chain, pharmacovigilance, regulatory correspondence, data integrity, investigator concentration, partner obligations and change-of-control restrictions.

8. Final go/no-go memorandum

GO