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CHMP Positive Opinions | February | 2024 | iPharmaCenter

Voydeya Received CHMP Position Opinion for Residual Haemolytic Anaemia for PNH

The CHMP of the European Medicines Agency (EMA) has recommended Voydeya (danicopan) for marketing authorization in the European Union (EU) as an adjunctive therapy to ravulizumab or eculizumab for adult patients grappling with paroxysmal nocturnal haemoglobinuria (PNH) and residual haemolytic anaemia. Voydeya, a pioneering oral Factor D inhibitor, has been developed to complement standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab), catering to the needs of approximately 10-20% of PNH patients encountering clinically significant extravascular haemolysis (EVH) despite treatment with a C5 inhibitor.

The positive opinion from CHMP stems from the conclusive findings of the pivotal ALPHA Phase III trial, with results from the 12-week primary assessment period published in The Lancet Haematology.

PNH stands as a rare and severe blood disorder characterized by intravascular haemolysis (IVH), leading to the destruction of red blood cells within blood vessels, alongside white blood cell and platelet activation, potentially triggering thrombosis (blood clots) and ensuing organ damage, and even premature death.

The ALPHA Phase III trial investigated Voydeya's efficacy and safety as an adjunct to Ultomiris or Soliris in PNH patients experiencing clinically significant EVH. Findings demonstrated Voydeya's success in meeting the primary endpoint of haemoglobin change from baseline to week 12, along with all key secondary endpoints, including transfusion avoidance and improvement in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.

Moreover, results from the ALPHA Phase III trial indicated Voydeya's overall tolerability, with no identification of new safety concerns. The most prevalent treatment-emergent adverse events observed during the trial included headache, nausea, arthralgia, and diarrhoea.

Voydeya has secured Breakthrough Therapy designation from the US Food and Drug Administration and PRIority MEdicines (PRIME) status from the EMA. Additionally, it has obtained Orphan Drug Designation in the US, EU, and Japan for PNH treatment. Recently approved in Japan, Voydeya's regulatory submissions are presently undergoing review in various other countries.


Janssen's Carvykti received CHMP position for early stages of relapsed and refractory multiple myeloma

Janssen has garnered a favorable nod from the Committee for Medicinal Products for Human Use (CHMP) concerning CARVYKTI (ciltacabtagene autoleucel; cilta-cel), signaling a notable breakthrough in its potential application for treating earlier stages of relapsed and refractory multiple myeloma (RRMM). This approval, rooted in insights from the Phase 3 CARTITUDE-4 study, suggests that cilta-cel holds promise for offering significant benefits to patients in initial treatment phases.

Today, the Janssen Pharmaceutical announced that the CHMP has endorsed a Type II variation for CARVYKTI (ciltacabtagene autoleucel) within the European Medicines Agency (EMA), specifically for the upfront treatment of RRMM. The proposed usage of cilta-cel targets adult RRMM patients who have undergone at least one prior therapy, including an immunomodulatory agent (IMiD) and a proteasome inhibitor (PI), displaying disease progression with the last treatment, and refractory to lenalidomide.

Notably, cilta-cel emerges as the pioneering chimeric antigen receptor T-cell (CAR-T) therapy to secure a positive CHMP verdict for this patient cohort, potentially even following the initial relapse. It represents an innovative CAR-T therapy directed at B-cell maturation antigen (BCMA), a protein abundantly expressed on myeloma cells.

CHMP's endorsement of cilta-cel finds robust support in data stemming from the CARTITUDE-4 study (NCT04181827), the first randomized Phase 3 investigation contrasting the efficacy and safety profiles of cilta-cel against alternative treatments such as pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd) in patients grappling with relapsed and lenalidomide-refractory multiple myeloma who have traversed one to three prior lines of therapy.

Presently, cilta-cel boasts conditional marketing authorization (CMA) for treating adults afflicted with RRMM post three prior lines of therapy.


Biogen's Qalsody received CHMP position opinion to treat amyotrophic lateral sclerosis

Biogen's QALSODY (tofersen) has received a favorable opinion from the Committee for Medicinal Products for Human Use (CHMP), marking a significant milestone in the treatment of a rare genetic form of amyotrophic lateral sclerosis (ALS).


SOD1-ALS, a profoundly debilitating and rare genetic variant of ALS, affects fewer than 1,000 individuals in Europe, making it an urgent area for medical intervention.


With QALSODY, Biogen has expanded our understanding of neurofilament's role in developing novel ALS therapies, potentially paving the way for further breakthroughs in this field.


Biogen has announced that the CHMP of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of QALSODY (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) linked to a mutation in the superoxide dismutase 1 (SOD1) gene. If approved by the European Commission (EC), QALSODY will be the first therapy authorized in the European Union specifically targeting a genetic cause of ALS, also referred to as motor neuron disease (MND).


The CHMP's recommendation for QALSODY is grounded in comprehensive evidence, encompassing its targeted mechanism of action, biomarker data, and clinical trial results. Results from the 28-week Phase 3 VALOR study demonstrated a remarkable 60% reduction in plasma neurofilament light chain (NfL) levels among participants receiving QALSODY compared to those in the placebo group, suggesting a decrease in neuronal damage. Additionally, participants treated with QALSODY exhibited trends toward improved physical functioning compared to those on placebo, as indicated by the ALS Functional Rating Scale-Revised (ALSFRS-R). Common adverse effects, occurring in 10% or more of patients treated with QALSODY and exceeding those in the placebo group, included pain, fatigue, fever, joint and muscle pain, and elevated levels of white blood cells and proteins in the cerebrospinal fluid. Serious neurological events, such as myelitis and/or radiculitis, papilledema with elevated intracranial pressure, and aseptic meningitis, have also been reported.


QALSODY (tofersen) is an antisense oligonucleotide (ASO) engineered to bind to SOD1 mRNA, reducing the production of SOD1 protein. The U.S. Food and Drug Administration (FDA) granted accelerated approval for QALSODY in the treatment of ALS in adults carrying a mutation in the SOD1 gene, based on observed reductions in plasma neurofilament light chain (NfL) levels in treated patients. Continued approval for this indication may be contingent upon confirming clinical benefits in further trials.



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