Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Epidermolysis Bullosa (EB) is a rare group of inherited disorders that typically manifest at
birth as blistering and lesion formation on the skin and, in some cases, the epithelial
lining of other organs, in response to little or no apparent trauma. In consequence, the skin
is extremely fragile which can result in shearing of the skin, causing a high risk of
infection. All forms of EB are both debilitating and life threatening. In some EB subtypes,
high mortality occurs before the age of 1 (Junctional Herlitz), and others in adolescences to
early adulthood, typically due to infection or failure to thrive. In addition, children
surviving into their 20's and 30's are also at risk for development of a virulent form of
squamous cell carcinoma, which is in many cases fatal.

There are no standard of care products available to treat the dermal manifestations of EB,
and there is no approved drug for EB in either Europe or the United States. There have been
numerous studies published on potential treatments for skin manifestations associated with
EB, including vitamin E therapy, systemic phenytoin, topical nonsteroidal agents,
cyproheptadine, tetracycline, and dapsone. No controlled studies showed clinical benefit of
any therapy. Newer exploratory treatments including skin grafts, bioengineered skin products,
and gene therapy have been unsuccessful to date.

This is an open label, multi-center extension study to assess the long-term safety of
topically applied ZORBLISA in patients with Simplex, Recessive Dystrophic, and Junctional
non-Herlitz Epidermolysis Bullosa. ZORBLISA will be applied topically, once a day, to the
entire body for a period of 1440 days.

Patients who successfully completed study SD-005 (on study drug at Visit 5) will have the
option to roll over into the SD-006 study. The baseline visit (Visit 1) will occur at Visit 5
for SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden
performed at the Visit 5 for SD-005 will be utilized as the baseline assessment for SD-006.
Patients will return for follow-up visits at months 1, 3, 6, 9 ,12, 15, 18, 21, 24, 27, 30,
33, 36, 39, 42, and 48 after Visit 1.

At each visit, assessments will include BSA of lesional skin and wound burden. For target
wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of
target wound area at the final visit for Study SD-005 will used as the baseline area size of
the target wound for SD-006. These unhealed target wounds from SD-005 will be assessed via
ARANZ at each subsequent scheduled visit until the target wound is documented as closed.
Closed wounds will be assessed for scarring.

Inclusion Criteria:

- Informed Consent form signed by the subject or subject's legal representative; if the
subject is under the age of 18 but capable of providing assent, signed assent from the
subject.

- Subject (or caretaker) must be willing to comply with all protocol requirements.

- Patients who completed the SD-005 study (on study drug at Visit 5).

Exclusion Criteria:

- Patients who do not meet the entry criteria outlined above.

- Pregnancy or breastfeeding during the study. (A urine pregnancy test will be performed
at the final visit for SD-005 for female patients of childbearing potential and
repeated at Visit 1 if these visits do no occur on the same day)

- Females of childbearing potential who are not abstinent or not practicing a medically
acceptable method of contraception