Catalyst Launches Proof-of-Concept Trial of Firdapse for SMA 3 Symptom Relief

Catalyst Launches Proof-of-Concept Trial of Firdapse for SMA 3 Symptom Relief

Catalyst Pharmaceuticals is launching a proof-of-concept Phase 2 trial of its investigational drug Firdapse (amifampridine phosphate), intended to relieve symptoms in ambulatory patients with spinal muscular atrophy (SMA) type 3.

If the treatment proves effective and safe in this group, which will include about 12 patients, Catalyst plans to continue the development of the drug while seeking orphan drug status with regulatory authorities — a designation that makes the development cheaper and faster.

The study will be led by Drs. Lorenzo Maggi and Giovanni Baranello at the Fondazione Istituto Neurologico Carlo Besta, a well-known referral center for SMA patients in Milan, Italy.

“We are very pleased that Drs. Maggi and Baranello and their team at the Fondazione Istituto Neurologico Carlo Besta have agreed to conduct this study for Catalyst of Firdapse in patients diagnosed with spinal muscular atrophy Type 3,” Patrick J. McEnany, CEO of Catalyst, said in a press release.

The trial will randomly assign patients to Firdapse or a control treatment. To get more robust results, patients will then switch treatments. Researchers will evaluate not only the safety of the treatment, but also its ability to improve symptoms.

Firdapse is a drug acting at the so-called neuromuscular junction — the spot at which a motor neuron connects to the muscle — by improving the signaling between a nerve and muscle.

Studies in both animal models and SMA patients show there are abnormalities in the neuromuscular junctions, seen before nerves start to degenerate and patients start developing symptoms. In fact, researchers believe that the changes in the nerve-muscle connection may contribute to the neurodegeneration.

Researchers have observed neuromuscular junction abnormalities in patients with SMA type 3 — a factor that contributed to Catalyst’s choice to focus on this patient group.

In addition to improving symptoms, researchers hope that the improved signaling at the neuromuscular junction may slow down the progression of neurodegeneration.

The trial will run through the first half of 2019.

“If the results from this trial support the safety and efficacy of Firdapse as a treatment for ambulatory patients with SMA type 3, we intend to submit an application for orphan drug designation and to pursue a clinical program required to obtain approval of Firdapse for this indication,” McEnany said.

One comment

  1. Dante says:

    In the 1990s, doctors in the US, on behalf of Muscular Dystrophy Association, approached a small family-owned manufacturer of active pharmaceutical ingredients in New Jersey, Jacobus Pharmaceuticals, about manufacturing amifampridine so they could test it in clinical trials. Jacobus did so, and when the treatment turned out to be effective, Jacobus and the doctors were faced with a choice — invest in clinical trials to get FDA approval or give the drug away for free under a compassionate use program. Jacobus elected to give the drug away, and did so for about twenty years.[13][14]

    In December 2015 a group of 106 neuromuscular doctors who had worked with both Jacobus and BioMarin/Catalyst published an editorial in the journal, Muscle & Nerve, expressing concern about the potential for the price of the drug to be dramatically increased should Catalyst obtain FDA approval, and stating that 3,4-DAPP represented no real innovation and didn’t deserve exclusivity under the Orphan Drug Act, which was meant to spur innovation to meet unmet needs.[12][31] Other commentators noted, however, that Jacobus was not able to meet the needs of all people who could benefit from the drug via its free program, and that its manufacturing practices had quality control problems, with 483 FDA citations in 2011 and 2012.[13] Similarly, at the urging of Assistance Publique-Hôpitaux de Paris, the French drug authority had reviewed the use of the free base in light of the manufactured phosphate form in 2006, and had urged doctors in France not to use the free base form due to quality issues.[32]

    Catalyst submitted its new drug application to the FDA and in February 2016 the FDA refused to accept it, on the basis that it wasn’t complete and in April 2016 the FDA told Catalyst it would have to gather further data.[33]

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