Portfolio cockpit

Adzynma

Launched
ADAMTS13 recombinant · Congenital thrombotic thrombocytopenic purpura
Recombinant ADAMTS13 enzyme replacement
Nov 2023 (US), 2024 (EU)
Launch readiness
+2 pts MoM
86
Regulatory
94
0
Trial momentum
62
0
Evidence
68
+2
Access / HTA
79
+2
Digital momentum
52
+1
Competitive
90
+3

Milestone rail

Nov 2023
FDA approval
Aug 2024
EC approval
2025
G-BA / HAS positive
2026
NICE managed access
2027
Prophylaxis label expansion (planned)

Clinical trials

ClinicalTrials.gov · Sep 2026
Active P3
1
Enrollment
72%
Completion in
26mo
Competing programs
1
Enrollment vs target72%

Publications

PubMed · Sep 2026
Last 12m
27
Growth
15%
+15%
Top-journal share
30%
Blood · 5JTH · 4ISTH abstracts · 9Other · 9

Regulatory

openFDA · Sep 2026
FDA approval Nov 2023Orphan DrugPriority Review
4 of 5 tracked milestones complete

HTA / access

HTA decisions / price databases · Sep 2026
NICE
positive
G-BA
positive
HAS
positive
CADTH
pending
PBAC
planned
List price (yr)
$480k
Class median
$350k
above class

Competitor landscape

Public pipelines · Sep 2026
RivalStatusLaunch inReadinessΔ efficacy
Plasma therapy
SoC (blood banks)
Marketed SoClaunched950 pts
—
No named rival
None disclosedlaunched00 pts
x: efficacy Δ vs SoC · y: access readiness

Risk / opportunity radar

Composite model · Sep 2026
CEO take
  • First and only recombinant ADAMTS13 — no direct competitor on horizon
  • Competitive position 90/100, best in portfolio
  • Ultra-orphan: digital footprint small (52/100) but community high-intent
  • EU HTA wins secured (G-BA, HAS positive); NICE managed access
  • Lifecycle: prophylaxis expansion and pediatric uptake

Digital signal layer

Multi-source listening · Sep 2026
Search interest index
Google Trends
38idx+4 pts
Congress abstracts 12m
ASCO / ASH / EHA / AAN / ACG
15abstracts+2 new
KOL share of voice
X / LinkedIn / podcasts
9%+1.8 pts
News & PR velocity
Press wires & trade media
8/mo+3 items
Patient community volume
Reddit / Inspire / patient forums
3.4k posts+0.6 k
Community sentiment
NLP over community posts
84/100+1 pt
Payer / formulary chatter
Payer bulletins / guidelines
24idx+5 pts