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EMA Drug Approvals | Europe drug approvals | 2024 | iPharmaCenter

Updated: Apr 23

Sl No







Myasthenia gravis




Transfusion-dependent anemia due to very low, low and intermediate-risk MDS

January 08, 2024

EC approved Rystiggo for gMG

UCB has received approval from the European Commission (EC) for Rystiggo (rozanolixizumab) as a supplementary element to conventional therapy for the management of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.

Rozanolixizumab, a humanized IgG4 monoclonal antibody with a 140 mg/ml solution for injection, attaches to the neonatal Fc receptor (FcRn), leading to decreased circulating IgG. This signifies validation as the primary therapy in Europe for adults with anti-AChR or anti-MuSK antibody-positive gMG, covering the two most common gMG subtypes.

The EC's choice ensues their earlier endorsement of UCB's Zilbrysq (zilucoplan) in December 2023 for managing adult patients with gMG who are anti-AChR antibody-positive. Zilucoplan, delivered once daily through subcutaneous injection, functions as a self-administered peptide inhibitor of complement component 5 (C5 inhibitor).

The EC validation for rozanolixizumab is validated by safety and effectiveness information from the pivotal Phase 3 MycarinG study (NCT03971422), disclosed in The Lancet Neurology in May 2023. The study exhibited statistically significant and clinically meaningful enhancements in gMG-specific outcomes compared to a placebo. Rozanolixizumab manifested effectiveness in the primary endpoint of the MG-ADL score at Day 43, indicating advancements in activities like breathing, talking, swallowing, and rising from a chair. Secondary effectiveness endpoints, comprising the Myasthenia Gravis Composite (MG-C) and the Quantitative Myasthenia Gravis (QMC) scores, further validated rozanolixizumab's efficiency. The MG-C score alteration from baseline to Day 43 unveiled a statistically significant contrast favoring rozanolixizumab over the placebo. Moreover, the QMG total score alteration from baseline to Day 43 displayed noteworthy enhancements with rozanolixizumab compared to the placebo.

Adverse reactions documented in the rozanolixizumab EU Summary of Product Characteristics involve headache (48.4%), diarrhea (25.0%), and fever (12.5%).

Generalized myasthenia gravis (gMG) is an uncommon, persistent autoimmune ailment influencing the neuromuscular junction, with a worldwide prevalence of 100–350 cases per every 1 million individuals.

This EC validation aligns with prior validations by the Japanese Ministry of Health, Labour and Welfare (MHLW) and the U.S. Food and Drug Administration (FDA) for rozanolixizumab and zilucoplan, correspondingly, in the treatment of gMG in grown-up patients.


April 02, 2024

European Union's Committee Enhances Bristol Myers Squibb’s Reblozyl (luspatercept) Approval for Primary Therapy of Transfusion-Dependent Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Bristol Myers Squibb made public that the European Commission (EC) has broadened the approval of Reblozyl (luspatercept) to encompass the primary treatment of adult individuals grappling with transfusion-dependent anemia attributable to very low, low, and intermediate-risk myelodysplastic syndromes (MDS). This endorsement of Reblozyl extends across all member states of the EU.

The endorsement is founded on the decisive Phase 3 COMMANDS trial, where Reblozyl showcased superior effectiveness when juxtaposed with epoetin alfa, an erythropoiesis stimulating agent, in the trial’s primary target of simultaneous independence from red blood cell transfusions and elevation in hemoglobin levels. Safety findings aligned with prior MDS investigations and were congruent with anticipated symptoms in this patient cohort. Reblozyl is also sanctioned in the United States and Japan for primary treatment of anemia linked with lower-risk MDS.

COMMANDS (NCT03682536) constitutes a Phase 3, open-label, randomized investigation evaluating the efficacy and safety of Reblozyl versus epoetin alfa for managing anemia associated with very low-, low- or intermediate-risk (IPSS-R) myelodysplastic syndromes (MDS) in individuals reliant on red blood cell (RBC) transfusions and who were naïve to erythropoiesis stimulating agents (ESA).

The primary parameter gauged in this investigation is RBC transfusion independence (RBC-TI) for 12 weeks with a mean hemoglobin (Hb) increase ≥1.5 g/dL. Principal secondary criteria encompass erythroid response (HI-E) of at least 8 weeks during weeks 1-24 of the study, RBC-TI ≥12 weeks, and RBC-TI for 24 weeks. Eligible participants were ≥18 years of age with lower-risk MDS necessitating transfusions. Participants were randomly allocated 1:1 to receive subcutaneous Reblozyl (commencing dose 1.0 mg/kg, titration up to 1.75 mg/kg) once every 3 weeks or epoetin alfa (commencing dose 450 IU/kg, titration up to 1050 IU/kg) weekly for ≥24 weeks.

At the time of the initial analysis (March 31, 2023), 363 participants were randomly assigned 1:1 to Reblozyl and epoetin alfa. Results from the primary analysis of the intent to treat (ITT) population showed:

  • 60.4% of participants on Reblozyl versus 34.8% (n=63) of participants on epoetin alfa achieved the primary parameter of RBC-TI lasting at least 12 weeks with concurrent mean Hb increase of at least 1.5 g/dL within the first 24 weeks.

  • HI-E increase of at least 8 weeks was achieved by 74.2% (n=135) of Reblozyl participants versus 53% (n=96) of epoetin alfa participants.

  • RBC-TI of at least 12 weeks was achieved by 68.1% (n=124) of Reblozyl participants versus 48.6% (n=88) of epoetin alfa participants.

  • The duration of response was 128.1 weeks (108.3-NE) for Reblozyl in participants who achieved TI for at least 12 weeks (achieved weeks 1-24) compared to 89.7 weeks (55.9-157.3) for epoetin alfa.

Safety findings were consistent with prior MDS investigations, and progression to acute myeloid leukemia and total fatalities were comparable between both groups of the investigation. The most prevalent treatment-emergent adverse events in at least 10% of participants comprised diarrhea, exhaustion, COVID-19, hypertension, dyspnea, nausea, peripheral edema, asthenia, dizziness, anemia, back pain, and headache. Rates of reported fatigue and asthenia showed a decline over time.


April 24, 2024

European Commission approved Carvykti for treating relapsed or refractory multiple myeloma

Carvykti, has been approved by the European Commission, for treating relapsed and refractory multiple myeloma. This treatment targets BCMA, offering hope to patients who have undergone at least one prior therapy. Its expanded indication suggests the potential for a treatment break as early as the first relapse.

The approval of Carvykti stems from the PHASE 3 CARTITUDE-4 trial. Results showed a significant 74% reduction in the risk of disease progression or death compared to standard treatments, validating its efficacy.

This approval extends to adult patients with relapsed and refractory multiple myeloma who have received prior therapies, including IMiD and PI, and are refractory to lenalidomide. Carvykti utilizes innovative CAR-T cell therapy, targeting BCMA, a protein highly expressed in myeloma cells.

The approval paves the way for Carvykti to be the first BCMA CAR-T therapy available in Europe for eligible patients, potentially transforming treatment paradigms.

The expanded indication is backed by positive findings from the Phase 3 CARTITUDE-4 study, which evaluated Carvykti's efficacy and safety in relapsed and lenalidomide-refractory multiple myeloma patients. Notably, patients who received Carvykti demonstrated a significantly lower risk of disease progression or death compared to those on standard treatment.

At a median follow-up of 15.9 months, patients treated with Carvykti experienced prolonged progression-free survival compared to standard treatment. The one-year estimated progression-free survival rate was notably higher in the Carvykti group, with a remarkable overall response rate and a substantial proportion achieving complete response or better.

Moreover, Carvykti demonstrated a higher overall minimal residual disease negativity rate, indicating its potential to achieve deeper responses. Survival outcomes also trended favorably towards Carvykti compared to standard treatment.

The safety profile of Carvykti was evaluated, with hematologic adverse events being the most common. However, the therapy's benefits outweighed the risks, highlighting its potential as a game-changer in treating relapsed and refractory multiple myeloma.


April 23, 2024

Voydeya approved by European Commission for paroxysmal nocturnal haemoglobinuria

Voydeya gains approval in the European Union as a supplementary treatment to ravulizumab or eculizumab for adults afflicted with the uncommon condition PNH, characterized by lingering haemolytic anaemia.

The ALPHA Phase III trial unveils Voydeya, a pioneering oral Factor D inhibitor, as an adjunct to Ultomiris or Soliris, showcasing enhanced haemoglobin levels and diminished anaemia and fatigue. Voydeya (danicopan) secures authorization in the European Union (EU) as an add-on to ravulizumab or eculizumab for managing adult patients with paroxysmal nocturnal haemoglobinuria (PNH) grappling with residual haemolytic anaemia.

Voydeya marks a breakthrough as an oral Factor D inhibitor designed as a complement to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab), aiming to meet the needs of approximately 10-20% of PNH patients confronting clinically significant extravascular haemolysis (EVH) while under C5 inhibitor treatment. The EC's approval was backed by outcomes from the pivotal ALPHA Phase III trial, with findings from the 12-week primary evaluation phase published in The Lancet Haematology.

The ALPHA Phase III trial assesses the efficacy and safety of Voydeya as an augmentation to Ultomiris or Soliris in PNH patients experiencing clinically significant EVH. Results demonstrate Voydeya's fulfillment of the primary endpoint, showcasing a change in haemoglobin from baseline to week 12, alongside meeting all key secondary endpoints, including transfusion avoidance and alterations in the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.

ALPHA, a pivotal global Phase III trial, structured as a superiority study, evaluates Voydeya's efficacy and safety as an adjunct to C5 inhibitor therapy Soliris or Ultomiris in PNH patients facing clinically significant EVH. In this double-blind, placebo-controlled, multiple-dose trial, patients are enrolled and randomly assigned to receive Voydeya or placebo (2:1) alongside ongoing Soliris or Ultomiris therapy for 12 weeks.

A predefined interim analysis is conducted once 63 randomized patients complete the 12-week primary evaluation period or discontinue treatment by June 28, 2022. At the 12-week mark, patients on placebo plus Soliris or Ultomiris transition to Voydeya plus Soliris or Ultomiris, while those on Voydeya plus Soliris or Ultomiris maintain this regimen for an additional 12 weeks. Patients completing both treatment phases (24 weeks) have the option to partake in a two-year long-term extension period, continuing to receive Voydeya alongside Soliris or Ultomiris. The open-label phase of the study is ongoing.

Voydeya (danicopan), a pioneering oral Factor D inhibitor, operates by selectively inhibiting Factor D, a vital complement system protein implicated in amplifying the complement system response. Dysregulated activation of the complement cascade prompts an overzealous response, leading the body to assail its own healthy cells. Voydeya earns Breakthrough Therapy designation from the US Food and Drug Administration and PRIority MEdicines (PRIME) status from the European Medicines Agency. Additionally, Voydeya garners Orphan Drug Designation in the US, EU, and Japan for treating PNH.

Voydeya secures approval in the US, EU, and Japan as adjunctive therapy to ravulizumab or eculizumab for specific adults grappling with PNH.


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