Through its recently issued press release, bluebird bio announced the FDA’s accelerated approval of the first therapy to slow the progression of adrenoleukodystrophy (ALD). The disease is the result of mutations in the ABCD1 gene which enable long-chain fatty acids to accumulate in the white matter of the spinal cord and brain. This, in turn, leads to the breakdown of the protective sheath surrounding nerve cells (myelin). The drug eli-cel provides copies of the ABCD1 gene which aids in the production of ALDP.
The drug was approved by a panel of advisers to the FDA in June of this year. The approval was based on the results of the open-label, single-arm studies called Starbeam ALD-102 and ALD-104 Phase 3.
MFD (metastasis-free survival) was analyzed starting with the onset of symptoms in patients who received eli-cel as well as patients who did not receive treatment. At the 24-month mark, patients receiving eli-cel had a 72% chance of metastasis-free survival as opposed to 43% for untreated patients.
The Drug is Named SKYSONA® (elivaldogene autotemcel), a/k/a eli-cel. The therapy is applicable to young boys with early, active cerebral adrenoleukodystrophy (CALD). It has also been confirmed that a clinical hold on eli-cel’s development program is no longer in effect.
Young boys ages four through seventeen are the primary targets for CALD. The disease causes neurologic decline that is irreversible. The symptoms of this devastating disease include cortical blindness, inability to communicate, feeding issues, incontinence, loss of voluntary movement, and wheelchair dependence. Early diagnosis is critical because without treatment over half of the patients will succumb to the disease within five years.
Effective options, until SKYSONA’s approval, were allogeneic stem cell transplants (SCT). The SCTs were associated with sometimes fatal consequences especially if a matched donor with the same tissue type could not be found. Therefore, a proper donor is usually the patient’s sibling.
The co-founder of ALD Alliance, Elisa Seeger, notes the accomplishments medical science has put forth on behalf of these young children with specific reference to the expanded screening of newborns. She further stated how no parent can or should withstand the agony of seeing their child die a little more every day.
One of the conditions of the SKYSONA approval is that bluebird will follow up with long-term clinical data submitted to the FDA. The company estimates that data from the LTF-304 study will be included as well as fifteen years of data from commercially classified patients who are treated in a clinical trial.
Bluebird expects that by the end of the year 2022, several U.S. Qualified Treatment Centers will have the product available as well as the Boston Children’s Hospital and the Philadelphia Children’s Hospital.
Grade 3 or 4 abnormalities that were reported included lymphopenia, neutropenia, leukopenia, thrombocytopenia, anemia, and hypokalemia.
SKYSONA has been approved under the FDA accelerated approval program which is a pathway that allows drugs for the treatment of life-threatening unmet medical needs.
SKYSONA Safety Information can be found here.