Ipsen’s drug Bvlay has gotten its second approval for itching (pruritis) in a rare liver disease, this time in Alagille syndrome. The drug had previously been approved for progressive familial intrahepatic cholestasis. Another indication may be on the way in the near future, as Ipsen is currently in late-stage testing for the drug in a childhood liver disorder.
The US Food and Drug Administration (FDA) relies on recommendations from its several advisory committees when weighing whether to approve a drug. However, Congress is placing extra scrutiny on the composition of these committees when debating orphan drugs, as laid out in an explanatory statement in the 2023 FY Consolidated Appropriations Act. Currently, they are expected to be populated with experts in the general field, but there is no provision to make sure rare disease experts are on the panel.
The UK’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending the approval of Upstaza, PTC Therapeutic’s gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, if approved, would cost £3 million and would be the only treatment for the ultra-rare disorder.
Avidity Biosciences’ treatment for a rare muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD) has been given orphan drug status by the US Food and Drug Administration (FDA). The drug, if approved, would be the first for the disease, which results in deterioration of muscle function beginning in the upper extremities.
The US Food and Drug Administration has granted Orphan Drug Designation (ODD) to GSK’s Benlysta (belimumab). The designation applies to the use of Benylsta in treating the interstitial lung disease (ILD) associated with systemic sclerosis (SSc), a rare autoimmune connective tissue disorder. The drug is currently approved for treating active lupus and lupus nephritis.
Policies regarding orphan drug designations for a group of rare genetic eye diseases called inherited retinal dystrophies are changing, according to the European Medicines Agency (EMA). The changes would target terminology regarding the categories of disorders that are covered under the orphan drug designation, allowing drugs to be submitted into one or more of four subgroups.
Taking place in Spain this year, the World Orphan Drug Congress, the leading conference for orphan drugs and rare diseases worldwide, takes place in Sitges, Spain, from 17-14 November. The conference will bring together professionals from healthcare, research, regulatory agencies, payer organizations, and other key decision-makers to one place to connect and share ideas.
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