Alnylam Pharmaceuticals Patient Finder Analysis

Ada Cockpit | March 2026

Executive Summary

Alnylam Pharmaceuticals is an RNAi therapeutics company with four approved rare disease drugs. This analysis evaluates Patient Finder suitability for each approved drug based on underdiagnosis rates, diagnostic delays, addressable markets, and company motivation.

Key Findings

Drug Indication Tier Fit Score Addressable Undiagnosed USA Ada Revenue Opportunity (5% Capture)
AMVUTTRA ATTR-CM (Cardiac Amyloidosis) 1 9/10 47,430 $585M per year
GIVLAARI Acute Hepatic Porphyria 2 7/10 726 $14.6M per year
OXLUMO Primary Hyperoxaluria Type 1 2 7/10 1,870 $18.2M per year
AMVUTTRA hATTR Polyneuropathy 3 6/10 1,805 $7.7M per year
ONPATTRO hATTR Polyneuropathy 3 2/10 250 $1.2M per year

Strategic Recommendation

PRIORITIZE AMVUTTRA (ATTR-CM) - Tier 1 opportunity with massive underdiagnosis (80-90%), perfect timing (March 2025 launch), and company's #1 strategic priority. Bundle with GIVLAARI and OXLUMO for rare disease diagnostic expansion package.

1. AMVUTTRA (vutrisiran) - ATTR-CM Indication

🎯 Pitch Hook (Tier 1, 9/10 Fit Score)

"Alnylam, you're sitting on a $2.3 billion ATTR-CM franchise, but 80-90% of eligible patients remain undiagnosed. Your March 2025 launch beat guidance by $800M, yet only 1.7% of heart failure patients are even tested for ATTR-CM.

Ada Patient Finder can surface the 47,000 undiagnosed US patients waiting in primary care. Our symptom checker identifies heart failure red flags (carpal tunnel history, spinal stenosis, unexplained HF) and navigates patients to cardiology for PYP scans.

At just 1% capture, that's $117M in annual Patient Finder fees and $1.2 billion in new AMVUTTRA revenue. This isn't marketing spend—it's market expansion that pays for itself.

Your 'Alnylam 2030' strategy needs diagnostic expansion to hit that 25% CAGR. We're the fastest path to those 47,000 US patients and 300,000+ globally. Let's talk."

Market Numbers

Metric USA DACH Rest of World
Total ATTR-CM Patients 91,260-106,860 40,222 540,000
Undiagnosed (80%) 73,008-85,488 32,178 432,000
Drug-Addressable Undiagnosed 47,430 19,259 258,552
Net Revenue Per Patient $238,000 $380,800 $380,800
Patient Finder Fee (10%) $23,800 $38,080 $38,080

Clinical Profile

Characteristic Details
Primary Symptoms Heart failure with preserved ejection fraction (HFpEF), dyspnea, fatigue, edema
Red Flags Carpal tunnel syndrome, lumbar spinal stenosis, atrial fibrillation
Diagnostic Delay 1.3 years from HF diagnosis; 3-6 years from symptom onset
Underdiagnosis Rate 80-90% (only 1.7% of HF patients tested)
Common Misdiagnoses Unspecified heart failure (32%), hypertrophic cardiomyopathy, aging-related heart disease
Ada Surface Ability 8/10 - High confidence identification

Opportunity Assessment

Scenario USA DACH ROW Total
At 1% Capture $11.3M $7.3M $98.5M $117M/year
At 5% Capture $56.4M $36.7M $492.4M $585M/year
Score Rating Rationale
Ada Surface Ability 8/10 Clear symptom profile (HF, dyspnea, fatigue), identifiable red flags
Company Motivation 10/10 Fastest-growing product, just launched, #1 strategic priority, aggressive expansion
Overall Fit Score 9/10 Massive underdiagnosis, severe delays, huge market, perfect timing

2. GIVLAARI (givosiran) - Acute Hepatic Porphyria

🎯 Pitch Hook (Tier 2, 7/10 Fit Score)

"GIVLAARI patients wait 15 years on average before diagnosis—longer than any other condition in your portfolio. That's 15 years of unnecessary suffering, ER visits, and misdiagnoses as IBS or endometriosis.

Ada can cut that delay by identifying the 726 undiagnosed US patients with the classic triad: severe episodic abdominal pain + neurologic symptoms + dark urine. We surface them from gastroenterology and primary care, where they're lost in the differential.

At $442K net revenue per patient, even 5% capture adds $14.6M in annual Patient Finder fees and $146M in new GIVLAARI revenue. This is a mature asset with 18% growth—patient finding can unlock the next growth phase.

The 15-year delay isn't a medical problem. It's a patient-finding problem. We solve it."

Market Numbers

Metric USA DACH Rest of World
Total Symptomatic AHP Patients 3,350 951 25,000
Undiagnosed (60%) 2,010 571 15,000
Drug-Addressable Undiagnosed 726 206 5,415
Net Revenue Per Patient $442,000 $460,000 $460,000

Clinical Profile

Primary Symptoms Severe episodic abdominal pain (90%), neurologic symptoms, psychiatric manifestations
Red Flags Dark/reddish urine during attacks, women of reproductive age
Diagnostic Delay 15 years average
Common Misdiagnoses IBS, IBD, appendicitis, endometriosis, psychiatric disorders
Ada Surface Ability 7/10 - Distinctive symptom constellation

Scores

Ada Surface Ability 7/10 Severe abdominal pain + neuro + psych is unique pattern
Company Motivation 6/10 Mature product, steady growth, not top priority but open to expansion
Overall Fit Score 7/10 15-year delay, high-value patients, clear symptoms

3. OXLUMO (lumasiran) - Primary Hyperoxaluria Type 1

🎯 Pitch Hook (Tier 2, 7/10 Fit Score)

"Half of adult PH1 patients reach end-stage kidney failure before diagnosis. That's preventable with early detection—but diagnosis takes up to 30 years.

OXLUMO can preserve kidney function if started early. The problem? 1,870 undiagnosed US patients are waiting in nephrology and urology clinics, misdiagnosed as 'idiopathic kidney stones.'

Ada can identify the high-risk cases: recurrent calcium oxalate stones starting in childhood, family history, normal calcium/uric acid. We surface them before ESRD.

At $375K net revenue per patient, even 5% capture adds $18.2M in annual Patient Finder fees and $182M in new OXLUMO revenue. More importantly, it prevents kidney transplants and saves lives.

Nedosiran is competing for the same patients. First to diagnose wins. Let's talk."

Market Numbers

Metric USA DACH Rest of World
Total PH1 Patients 4,685 509 11,800
Undiagnosed (70%) 3,280 356 8,260
Drug-Addressable Undiagnosed 1,870 203 4,708
Net Revenue Per Patient $375,000 $600,000 $600,000

Clinical Profile

Primary Symptoms Recurrent kidney stones (79% of adults), nephrocalcinosis, hematuria
Red Flags Childhood onset, family history, calcium oxalate stones with normal calcium/uric acid
Diagnostic Delay 1.2 years (children) to 30 years (adults)
Catastrophic Outcome 52% at ESRD before diagnosis
Ada Surface Ability 6/10 - Recurrent stones identifiable, ultra-rare differential

Scores

Ada Surface Ability 6/10 Recurrent kidney stones, especially pediatric onset with family history
Company Motivation 6/10 Mature product, competition from nedosiran, some urgency
Overall Fit Score 7/10 Catastrophic delays (1-30 years), 52% at ESRD, preventable kidney failure

4. Lower Priority Drugs

AMVUTTRA (hATTR-PN) - Tier 3, 6/10 Fit Score

Position: Natural complement to ATTR-CM. Lead with ATTR-CM (Tier 1), add hATTR-PN as expansion.

Opportunity: 1,805 addressable undiagnosed US patients, $7.7M at 5% capture.

Challenge: Better diagnosed than ATTR-CM (40% vs 80-90% underdiagnosis), less distinctive symptoms (polyneuropathy vs cardiac).

ONPATTRO (patisiran) - Tier 3, 2/10 Fit Score

DO NOT PURSUE - Being actively cannibalized by AMVUTTRA. Revenues down 32% in 2025 (Q4: $32M). Alnylam is transitioning patients to AMVUTTRA (subcutaneous vs IV, better dosing).

Company motivation: 1/10 - Zero appetite for ONPATTRO patient finding. Redirect to AMVUTTRA if approached.

Strategic Recommendations

Priority 1: PURSUE AMVUTTRA (ATTR-CM)

Priority 2: BUNDLE GIVLAARI + OXLUMO

Priority 3: CONSIDER hATTR-PN AS ADD-ON

Priority 4: SKIP ONPATTRO

Total Opportunity Summary

Tier Drugs Addressable Undiagnosed USA Revenue Opportunity (5% capture)
Tier 1 AMVUTTRA (ATTR-CM) 47,430 $585M/year
Tier 2 GIVLAARI + OXLUMO 2,596 $32.8M/year
Tier 3 AMVUTTRA (hATTR-PN) 1,805 $7.7M/year
TOTAL (excluding ONPATTRO) 51,831 $625.5M/year

Pipeline Notes

No Phase 3+ candidates as of March 2026. Three early-stage programs in Phase 1/2:

Trial Finder opportunity: ALN-HTT02 (Huntington's Phase 1 enrollment)