The therapy’s approval stems from its ability to restore dopamine synthesis in the brain, enabling children and adults with AADC deficiency to achieve critical motor development milestones. “We look forward to bringing this transformational gene therapy to patients in the United States,” added Klein, highlighting their preparation efforts, including establishing centers of excellence and training surgeons in the delivery procedure.
KEBILIDI™, a one-time gene replacement therapy, is administered directly to the putamen of the brain through stereotactic neurosurgery. Clinical trials have demonstrated significant improvements in motor function and overall quality of life for patients. This FDA nod was based on data from an ongoing global study, with confirmatory evidence to be gathered from long-term patient follow-ups.
AADC deficiency, often fatal and profoundly disabling, leaves children unable to synthesize dopamine, causing severe motor dysfunction, oculogyric crises, and life-threatening complications. With its approval, KEBILIDI offers hope to families affected by this condition, positioning PTC Therapeutics at the forefront of neurologic innovation.
Sanofi and Regeneron’s DUPIXENT sBLA Accepted by FDA for Chronic Spontaneous Urticaria
The FDA has accepted the resubmission of DUPIXENT’s (dupilumab) sBLA for treating chronic spontaneous urticaria (CSU) in patients aged 12 and older whose condition is inadequately managed with antihistamines. If approved, DUPIXENT would become the first targeted therapy for CSU in over a decade, with a decision expected by April 18, 2025. The resubmission is backed by pivotal data from the LIBERTY-CUPID Phase 3 trials, showing significant reductions in itch and hive activity among patients on standard antihistamine treatments.
Dr. George Yancopoulos, President and Chief Scientific Officer at Regeneron, highlighted the significance of the submission, stating, “Patients with inadequately controlled CSU often suffer debilitating symptoms. These pivotal results reinforce DUPIXENT’s potential as a transformative option for this population.” Similarly, Dr. John Reed, Global Head of R&D at Sanofi, noted, “This milestone underscores our commitment to expanding DUPIXENT’s impact across allergic and inflammatory diseases.”
The LIBERTY-CUPID clinical program demonstrated DUPIXENT’s efficacy across multiple studies, with Study C confirming significant reductions in itch and hive activity, consistent with earlier results from Study A. Safety data were generally in line with DUPIXENT’s established profile, with common side effects including injection site reactions and mild COVID-19 infections.
Currently approved for multiple indications, DUPIXENT’s potential expansion into CSU marks a critical step in addressing the unmet needs of more than 300,000 people in the U.S. who live with the burden of inadequately controlled CSU. If approved, this therapy could redefine care standards for the condition.
CHMP Recommends TAGRISSO for Unresectable EGFR-Mutated Lung Cancer
AstraZeneca’s TAGRISSO (osimertinib) has received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the treatment of adults with unresectable, locally advanced EGFR-mutated non-small cell lung cancer (NSCLC) following platinum-based chemoradiation therapy (CRT). The endorsement is backed by groundbreaking findings from the LAURA Phase III trial, which showed that TAGRISSO extended median progression-free survival (PFS) to 39.1 months—a significant improvement compared to 5.6 months with placebo. These results reflect an 84% reduction in disease progression or death.
Dr. Manuel Cobo, an investigator in the trial, emphasized the importance of the findings, stating, “The LAURA results build on the established efficacy of osimertinib and support the approval of the first targeted therapy for patients with unresectable, EGFR-mutated lung cancer.” Similarly, Susan Galbraith, AstraZeneca’s EVP for Oncology R&D, remarked, “Today’s news reinforces TAGRISSO as the backbone therapy in EGFR-mutated non-small cell lung cancer… representing a pivotal step in transforming care for patients.”
TAGRISSO’s safety profile in the LAURA trial aligns with its established tolerability, with no new safety concerns reported. This EU recommendation follows TAGRISSO’s recent approval for similar indications in the U.S. and highlights its expanding global footprint, with regulatory reviews underway in China, Japan, and other countries.
With more than 450,000 lung cancer diagnoses annually in Europe and approximately 10-15% of NSCLC patients harboring EGFR mutations, TAGRISSO addresses a critical unmet need in a significant patient population. Already approved in over 100 countries for a range of EGFR-mutated NSCLC stages, including early-stage and metastatic settings, TAGRISSO continues to solidify its role as a leading therapy in lung cancer care.
FDA Approves DANZITEN for Chronic Myeloid Leukemia
Azurity Pharmaceuticals has announced the FDA approval of DANZITEN™, a groundbreaking formulation of nilotinib indicated for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP) and for those with CP or acute phase (AP) resistant or intolerant to prior imatinib therapy. Unlike its predecessor, TASIGNA®, DANZITEN eliminates mealtime restrictions, offering a more patient-friendly treatment option without compromising efficacy.
Highlighting the advancement, Azurity CEO Richard Blackburn stated, “DANZITEN offers a new nilotinib treatment option with the equivalent efficacy to Tasigna but without the fasting requirements, liberating CML patients from mealtime restrictions.” This improvement addresses the fasting-related variability in Tasigna’s bioavailability, which, when improperly taken with food, poses a risk of cardiotoxicity by prolonging the QT interval on ECG.
DANZITEN leverages a re-engineered formulation of nilotinib, ensuring consistent pharmacokinetics regardless of fasting state or meal type. With a lower dose, it achieves equivalent efficacy to Tasigna, delivering the deep molecular responses critical for treatment-free remission in some patients. By removing the fasting requirements, DANZITEN may enhance patient adherence, a crucial factor in achieving optimal outcomes.
Set to launch in the coming weeks, DANZITEN will be available via Biologics by McKesson. Azurity also introduced the DANZITENCONNECT™ program, offering support such as prior authorization assistance, co-pay reductions, and free initial treatment, easing accessibility for eligible patients.
Syndax Gains FDA Approval for REVUFORJ in Acute Leukemia
The FDA has approved Revuforj® (revumenib), the first menin inhibitor, for treating relapsed or refractory (R/R) acute leukemia with KMT2A translocations in patients aged one year and older. The approval, granted under the FDA’s Real Time Oncology Review (RTOR) program, marks a breakthrough for a condition with limited options and poor prognosis.
Michael A. Metzger, CEO of Syndax, highlighted the milestone, stating, “This approval reflects the dedication of our team and the patients and clinicians who participated in our trials. We are prepared to launch Revuforj this month and advance its development across the treatment continuum for KMT2A-rearranged acute leukemias.”
Revuforj’s approval is based on the Phase I/II AUGMENT-101 trial, which showed a 21% complete remission rate (CR+CRh) in 104 patients, with a median remission duration of 6.4 months. Nearly a quarter of patients proceeded to stem cell transplantation, offering hope in a disease where conventional therapies often result in relapse and poor survival rates.
Dr. Ghayas C. Issa of MD Anderson Cancer Center emphasized the therapy’s significance: “This approval is a major breakthrough for patients with R/R acute leukemia with KMT2A translocation, a genetic alteration associated with a poor prognosis. Revuforj’s robust efficacy represents a substantial improvement over previously available therapies.”The drug will be available in 110 mg and 160 mg tablets starting in November through specialty distributors. A 25 mg tablet, catering to patients under 40 kg, is expected by early 2025, with an oral solution available in the interim. Syndax is also launching SyndAccess™, a support program offering financial assistance and personalized resources to ensure patient access.
