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Designing One Clinical Trial for Two Regulators: FDA and PMDA
Glafabra
Designing a trial for two regulators at once is harder than designing it for one — but it's cheaper than doing it twice.
Glafabra's U.S. Phase 1/2 protocol is being built from the outset to generate the specific biomarker package — alpha-Gal A enzyme activity, plasma lyso-Gb3, eGFR slope — that PMDA would require for a Japan submission. That's a deliberate choice made now, months before IND filing...more
Meet Glafabra's Acting CFO, Sidney Norton
Meet Sidney (Sid) Norton, Acting Chief Financial Officer.
Sid brings 25 years across private, government, and not-for-profit healthcare finance. He currently serves as CFO of Intermountain Primary Children's Hospitals, and previously held senior financial and operational roles at Cincinnati Children's, including VP of the Cancer and Blood Diseases Institute, plus five years in system finance at In...more
What Percentage of Fabry Patients Can Take Oral Therapy? — DECODE No. 15
(Answer to the question last time on August 20: lupus, multiple sclerosis, and other conditions with overlapping symptoms are common Fabry misdiagnoses.)
There's an oral therapy for Fabry disease, but it only works for patients whose specific GLA variant responds to it.
Roughly what percentage of Fabry patients carry a variant that qualifies for oral therapy? Drop your guess in the comments below.
...more
Six Weeks Into Testing the Waters: Glafabra's Progress Update
Six weeks into Testing the Waters. Here's where things actually stand.
FDA INTERACT meeting: done. IND protocol: in development. University of Utah Health: planned to be the primary clinical site. DECODE has answered 13 real questions about Fabry disease so far, and our LinkedIn Tuesdays are now team introductions.
If you've been reading since Day 1 — thank you. If you just found us — welcome, and...more
Ask Glafabra Anything: Live Q&A on the BioTech Funding Portal
This portal is a live Q&A, not a static page.
Questions we've gotten recently: Why did Glafabra choose melphalan over busulfan for conditioning? What happens to the corrected cells over the long term? Why is University of Utah Health planned to be the clinical site?
We answer every one publicly so the whole community benefits from the answer, not just the person who asked.
What's your question...more
How Outpatient Conditioning Enabled Same-Day Discharge in the FACTS Trial
Same-day discharge isn't a footnote. For a patient and their family, it's the difference between one afternoon and a hospital stay.
Four of five FACTS trial patients received their conditioning regimen and went home the same day — because it used a gentler, non-myeloablative approach instead of bone-marrow-transplant-intensity chemotherapy. Glafabra built Live-cel around that outpatient approach f...more
Beyond Activation: Why Building Brown Fat May Succeed Where Waking It Up Failed
Beyond Activation: Why Building Brown Fat May Succeed Where Waking It Up Failed
In the late 1980s and throughout the 1990s, several large pharmaceutical companies tried to develop obesity drugs that would acutely activate thermogenesis - the body’s process of dissipating energy as heat. The idea was to increase resting metabolic rate and turn stored fat into heat by stimulating the activity ...more
What Conditions Is Fabry Disease Often Mistaken For? — DECODE No. 14
(Answer to the question last time on August 17: women — X-inactivation can leave female patients with near-normal enzyme levels on a standard blood test, so genetic testing of the GLA gene is the reliable way to diagnose them.)
Fabry's symptoms touch so many organ systems that it regularly gets mistaken for other conditions entirely — sometimes for years.
Name one condition Fabry disease is common...more
