Indication intelligence, analysis and sector perception

bluebird bio (BLUE) announced the FDA has granted Accelerated Approval of SKYSONA® (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystro

September 21, 2022 8:47pm

A second approval ...

Also the largest volume of three (3) session: bluebird bio (BLUE) +9.118 million shares traded (-$0.69) after Tuesday’s +21.503 million shares traded (+$0.93) after Monday’s -30.962 million share traded (-$0.41 or -6.47%) – EXPECT an offering!

BLUE also confirmed that the previous clinical hold on the eli-cel clinical development program has been lifted.

Therapy pricing is an issue...

CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.

Nearly half of patients who do not receive treatment die within five years of symptom onset.

Prior to the approval of SKYSONA treatment, effective options were limited to allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with the risk of serious potential complications including death, that can increase dramatically in patients without a human leukocyte antigen (HLA) matched donor.

CALD strikes young boys in the prime of their development, robbing them of core neurologic functions necessary for survival.

The Bottom Line: questionable issue … cost, cost and again cost of treatment and POTENTIAL of reimbursement is a serious question for those in need?

BLUE anticipates that commercial product will be available by the end of 2022 through a limited number of Qualified Treatment Centers (QTCs) in the United States, including Boston Children’s Hospital and Children’s Hospital of Philadelphia.

BLUE has set the wholesale acquisition cost of SKYSONA in the U.S. at $3 M.

35 companies, 1 interpreter!

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