Ann: New Indication for ATL1102 Limb Girdle Muscular Dystrophy R2, page-36

If the EU DMD trial goes well (it's shown to be safe and effective), whether some LGMD patients might be able access ATL1102 early, outside of a trial setting ---- I assume we have an approved drug.- Safe and efficacy

Not sure the attached FDA document can provide some hints... just take a look and the scenario given.... and of course... This is only for our general knowledge!

https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label

Unapproved use of an approved drug is often called “off-label” use. This term can mean that the drug is:

• Used for a disease or medical condition that it is not approved to treat, such as when a chemotherapy is approved to treat one type of cancer, but healthcare providers use it to treat a different type of cancer.
• Given in a different way, such as when a drug is approved as a capsule, but it is given instead in an oral solution.
• Given in a different dose, such as when a drug is approved at a dose of one tablet every day, but a patient is told by their healthcare provider to take two tablets every day.

If you and your healthcare provider decide to use an approved drug for an unapproved use to treat your disease or medical condition, remember that FDA has not determined that the drug is safe and effective for the unapproved use.

Questions you may want to consider:
Example:

• What is the drug approved for?
• What scientific studies are available to support the use of this drug to treat my disease or medical condition?
• What are the potential benefits and risks of treating my disease or medical condition with this drug?
• Are there any clinical trials studying the use of this drug for my disease or medical condition that I could enroll in..

From EMA: (I think they should have the same point of view as FDA??)
Definition of Off-label use ----Use of a medicine for an unapproved indication or in an unapproved age group, dosage, or route of administration.