ALD News: September 2026
Here are three ALD developments from September 2026. Each one includes who it applies to and a link to the original source.
Europe approves a first drug treatment for boys with cerebral ALD
What happened: The European Commission approved NEZGLYAL (leriglitazone) on September 21, 2026. It is the first approved drug treatment for cerebral ALD in the EU. minoryx
Who it’s for: Boys ages 2 to 12 with cerebral ALD who have gadolinium-negative (non-enhancing) brain lesions on MRI and a neurological function score (NFS) of 0 or 1.
Why it matters: It is taken by mouth once a day. Until now, there was no approved drug treatment for early intervention.
Where it stands: The approval covers the 27 EU countries, plus Norway, Iceland, and Liechtenstein. The first launch is expected in Germany by the end of 2026. Other launches depend on national reimbursement talks. The company is still working toward U.S. approval. Talk with your child’s care team about what this means for your family.
Learn more: Press release from Minoryx and Neuraxpharm
The adult Phase 3 trial (CALYX) is now fully enrolled
What happened: The last patient was enrolled in the CALYX trial on September 9.
Who's in it: 41 adult men with cerebral ALD who have gadolinium-enhancing brain lesions. Stem cell transplant was not recommended for them or was declined.
Why it matters: The trial compares leriglitazone to a placebo. It is designed to test whether the drug is safe and effective in adults.
Where it stands: Phase 3. First results are expected in early 2028. The company says the FDA has agreed this trial can support a U.S. application
Learn more: Press release from Minoryx and Neuraxpharm
A bill to help states add recommended newborn screenings
What happened: Rep. Nick Langworthy announced the introduction of the bipartisan Surge to Save Newborns Act on September 25. Rep. Kim Schrier co-led it.
Who it affects: Newborns and their families. ALD has been on the federal list of recommended newborn screenings since 2016.
Why it matters: Adding a condition to the federal list does not mean every state starts screening for it right away. The bill would create a federal grant program to help states put recommended screenings into practice. It would provide $35 million a year from fiscal year 2027 through 2031.
Where it stands: Introduced in the House. It still needs to pass both chambers and be signed into law. Cure ALD supports this bill.
Learn more:
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