@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
GCC IGA NEPHROPATHY Competitive Intelligence
GCC IGA NEPHROPATHY Competitive Intelligence
Fewer than 20 GCC centres perform the kidney biopsy IgA nephropathy diagnosis requires, gating an 8,000-12,000 patient market.
Targeted-release budesonide...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
US HEREDITARY ANGIOEDEMA Competitive Intelligence
US HEREDITARY ANGIOEDEMA Competitive Intelligence
The HAE prophylaxis class is splitting by route: one oral agent against four injectables, plus 2025's first oral on-demand treatment.
HAE prophylaxis has moved...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
UK HEREDITARY ANGIOEDEMA Competitive Intelligence
UK HEREDITARY ANGIOEDEMA Competitive Intelligence
NICE has cleared three HAE prophylaxis therapies since 2019, but donidalorsen and sebetralstat remain without a confirmed NHS funding decision.
NICE has issued...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
GCC HEREDITARY ANGIOEDEMA Competitive Intelligence
GCC HEREDITARY ANGIOEDEMA Competitive Intelligence
More than 85% of GCC's estimated 1,200-1,500 HAE patients remain undiagnosed despite lanadelumab's 87% attack-rate reduction.
C1-inhibitor (Berinert) is the...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
US GAUCHER DISEASE Competitive Intelligence
US GAUCHER DISEASE Competitive Intelligence
Eliglustat is Gaucher's only first-line oral therapy, cutting spleen volume 30% versus placebo in ENGAGE, at an annual WAC of $310,250.
Gaucher disease type 1...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
US FABRY DISEASE Competitive Intelligence
US FABRY DISEASE Competitive Intelligence
Pegunigalsidase now challenges Fabrazyme's two-decade lead, while oral migalastat works for only 35 to 50% of Fabry patients.
Fabry disease is an X-linked...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
UK FABRY DISEASE Competitive Intelligence
UK FABRY DISEASE Competitive Intelligence
Pegunigalsidase's TA915 recommendation gives NICE a third Fabry option for the 80-120 UK patients failing standard ERT.
NHS Highly Specialised Services commission...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
GCC FABRY DISEASE Competitive Intelligence
GCC FABRY DISEASE Competitive Intelligence
Migalastat is covered for 35-50% of GCC Fabry patients with an amenable mutation, but fewer than 15% have been tested.
Classic Fabry disease prevalence in GCC is...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
US DRAVET SYNDROME Competitive Intelligence
US DRAVET SYNDROME Competitive Intelligence
Fenfluramine cut convulsive seizures 62.3% more than placebo, but its REMS burden and 3x price premium over cannabidiol define the real contest.
Dravet syndrome's...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
UK DRAVET SYNDROME Competitive Intelligence
UK DRAVET SYNDROME Competitive Intelligence
A new Dravet entrant must beat an entrenched three-drug NHS sequence, not just clear NICE's cost bar.
NHS England Highly Specialised Services commission Dravet...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
GCC DRAVET SYNDROME Competitive Intelligence
GCC DRAVET SYNDROME Competitive Intelligence
A narcotics-law barrier blocks cannabidiol in the GCC, making stiripentol, with a 71% adjunct responder rate, the de-facto standard instead.
Cannabidiol...
@moatrx
• 3 weeks ago
• comments: 0
Posted a new blog:
US COPD Competitive Intelligence
US COPD Competitive Intelligence
Two 2024 approvals, ensifentrine and dupilumab, ended a decade of inhaler-only competition in the US COPD maintenance market.
Through 2023 the US COPD maintenance...
