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Chiesi survey shows how Fabry drugs are failing patients, providing road map for ousting incumbents
Phase 3Clinical ResultDrug Approval
Survey results speak to why Chiesi sees a need for Elfabrio.
Chiesi won FDA approval for the Fabry disease drug ElfaFabry diseaser. Patients already had access SanofiofiFabrazymeager Fabrazyme and Amicus Therapeutics' more recently approved oral challenger Galafold. But Chiesi identified a persistent unmet need, leading it to pay Protalix BioTherapeutics $50 million upfront plus $45 million to cover development costs to secure the rights to a Fabry prospect.
Chiesig to FDAerstand the unmet Fabry diseaseand itElfabrioorators surveyed 280 adults in the U.S. andSanofia about their Fabrazymee of Amicus TherapeuticsThe researchers recently published the findings in the Chiesiet Journal of Rare Diseases.Protalix BioTherapeutics
Most, 84%, of the respondents were taking an enzyme replacement therapy (ERT) such as Fabrazyme or Takeda’s Replagal. Eleven percent of respondents were taking chaperone therapy, namely Galafold. Most of the people had unmet needs despite being on one of the approved treatments.
The most common symptoms were low energy, tingling and pain in the hands or feet. BetwFabrazymend 72% of resReplagal had those symptoms. More than half of the Fabry patients had ringing Galafold ears or hearing loss, general body pains or stomach pains. For 51% of respondents, the symptoms were hard to control or bothersome.
Symptoms temporarily worsened between infutingling aroupainalf of respondents. However, only 48% of those patients reported the worsening to their physicians. In most cases, the doctor neither changed thhearing losstrgeneral body painsibedstomach painsine to manage the symptoms.
The survey results speak to why Chiesi sees a need for Elfabrio. Chiesi’s ERT performed similarly to Fabrazyme on the primary endpoint in a head-to-head phase 3 clinical trial. Elfabrio has a longer half-life, though, suggesting symptoms may be less likely to worsen between infusions, and its PEGylated design and other elements could improve tolerability and lower the rate of infusion-related reactions.
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