Drug delivery system to double eligible patients for SMA treatment

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Noipornpan)
(Image: Getty/Noipornpan)

Related tags: Spinal muscular atrophy, Biogen, Spinraza

Researchers from the Clinic for Special Children have developed a drug delivery catheter system that could reach a larger population of patients receive Biogen’s Spinraza treatment.

Clinicians and researchers working together in Strasburg, Pennsylvania, have trialled an intrathecal catheter system (SIC) that is able to deliver Spinraza (nusinersen) directly into the cerebrospinal fluid in patients with spinal muscular atrophy (SMA).

The newly developed system functions by connecting an intrathecal catheter to an implantable infusion port.

Through the use of the port, the therapy was able to be delivered to patients at a reduced cost and within an administration time of 20 minutes.

In the current form of drug delivery, Spinraza is given to patients via a lumbar puncture every four months.

Researchers noted that this can be difficult, “the majority of surviving SMA patients have skeletal deformities or spinal hardware that make it difficult to safely and reliably access the cerebrospinal fluid.”

In addition, the study​ cited a rate of 20-50% of newborns or young children having traumatic complications due to the current form of delivery.

As a result, the new form of drug delivery could double the number of patients eligible for treatment and reduce the cost of administration by five- to ten-fold. The next step for this drug delivery method is to expand into larger clinical trials.

At present, Spinraza is the only available disease-modifying treatment for SMA and is a major driving force behind Biogen’s revenues​ – bringing in $423m (€365m) in the second quarter of 2018, globally.

SMA is a genetic condition in which muscles become progressively weaker with age, which can cause significant problems with any muscle-related activity, such as breathing or swallowing.

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