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The FDA cleared four novel drugs in March across hepatology, dermatology, rare pediatric lysosomal disease, and gynecologic oncology. Each is tightly placed, from PBC-associated itch to platinum-resistant ovarian cancer.
Lynavoy: Alfasigma takes over a newly approved PBC itch asset from GSK
On March 17, the FDA approved Lynavoy (linerixibat), a GSK-developed ileal bile acid transporter inhibitor, for cholestatic pruritus associated with primary biliary cholangitis in adults. Days earlier, GSK agreed to transfer worldwide rights to Alfasigma, positioning the Italian liver-focused company to commercialize the first U.S.-approved drug specifically for PBC-associated itch.
Linerixibat is an ileal bile acid transporter inhibitor designed to reduce bile acid reuptake in the gut. The strategic point is direct symptom biology: Lynavoy targets a recognized driver of cholestatic itch rather than relying on repurposed agents borrowed from adjacent settings.
Approval was supported by the phase 3 GLISTEN trial, which showed improvement in worst-itch scores and itch-related sleep disruption versus placebo. Tolerability was shaped mainly by gastrointestinal events, especially diarrhea and abdominal pain.
Icotyde: Johnson & Johnson brings oral IL-23 pathway inhibition into psoriasis
Also on March 17, the FDA approved Icotyde (icotrokinra), with Janssen Biotech as sponsor, for moderate-to-severe plaque psoriasis in adults and in patients aged 12 years and older weighing at least 40 kilograms who are candidates for systemic therapy or phototherapy. For Johnson & Johnson, the approval puts validated IL-23 biology into a once-daily oral format rather than an injectable one.
Icotrokinra is an oral peptide that targets the IL-23 receptor. In commercial terms, that gives Johnson & Johnson a convenience-based angle in a market where biologics have set the efficacy benchmark but route of administration still shapes uptake.
The approval was backed by the phase 3 ICONIC program, including ICONIC-LEAD and ICONIC-ADVANCE. Johnson & Johnson reported strong week 16 skin clearance outcomes, including PASI 90 and Investigator’s Global Assessment responses, with no major new safety signals reported during the controlled period.
Avlayah: Denali secures a CNS-directed Hunter syndrome approval
Denali Therapeutics received accelerated approval on March 25 for Avlayah (tividenofusp alfa-eknm) for the treatment of neurologic manifestations of Hunter syndrome iin presymptomatic or symptomatic pediatric patients weighing at least 5 kilograms, prior to advanced neurologic impairment. This was the month’s most technically differentiated approval because it addresses the neurologic component of MPS II, a long-standing limitation of standard enzyme replacement therapy.
Avlayah is an enzyme replacement therapy engineered to cross the blood-brain barrier through transferrin receptor-mediated transport. That design is the product’s defining feature: peripheral enzyme activity was never the full problem in Hunter syndrome when CNS decline drives irreversible morbidity.
The accelerated approval was based on reduction in cerebrospinal fluid heparan sulfate, a surrogate biochemical endpoint, from an open-label phase 1/2 study. Continued approval depends on confirmatory evidence from the ongoing COMPASS trial. Safety is infusion-centered and includes a boxed warning for hypersensitivity, including anaphylaxis.
Lifyorli: Corcept adds a resistance-focused option in ovarian cancer
Later the same day, the FDA approved Lifyorli (relacorilant) from Corcept Therapeutics, in combination with nab-paclitaxel, for adults with platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer after one to three prior systemic regimens, including bevacizumab. In a difficult post-bevacizumab setting, the approval adds a mechanistically distinct partner rather than another conventional cytotoxic permutation.
Relacorilant is a selective glucocorticoid receptor antagonist intended to blunt cortisol-linked tumor survival signaling and improve chemotherapy sensitivity. The mechanism targets glucocorticoid-driven resistance pathways to enhance response to a standard chemotherapy backbone.
Approval was supported by the phase 3 ROSELLA study in 381 patients, which showed statistically significant improvements in progression-free and overall survival versus nab-paclitaxel alone. Safety was consistent with a combination oncology regimen, including hematologic toxicity, gastrointestinal events, fatigue, rash, and adrenal insufficiency risk.
The Strategic Brief
Lynavoy and Icotyde are the market-shaping approvals. Lynavoy establishes a defined U.S. category in PBC-associated cholestatic pruritus, while Icotyde brings oral IL-23 pathway inhibition into psoriasis.
Avlayah is the focused orphan launch. It extends enzyme replacement into the central nervous system in Hunter syndrome, supporting a neurologic treatment claim.
Lifyorli is the tactical oncology addition. It introduces glucocorticoid receptor antagonism as a resistance-modulating strategy in platinum-resistant ovarian cancer, supported by overall survival benefit.
Sources
Lynavoy: FDA approval letter; GSK press release; Alfasigma press release.
Icotyde: FDA approval letter; Johnson & Johnson press release.
Avlayah: FDA press announcement; Denali Therapeutics press release.
Lifyorli: FDA press announcement; FDA approval letter; Corcept Therapeutics press release.
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