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CHMP Delivers Positive Opinions on Medications in April 2024

The recent meeting of the Committee for Medicinal Products for Human Use (CHMP), held from April 22 to April 25, 2024, has resulted in a series of positive opinions on various medications, encompassing a range of therapeutic areas and treatment modalities. These opinions signal potential advancements in healthcare and offer renewed hope for patients facing a multitude of medical conditions.

Altuvoct (Efanesoctocog Alfa) for Hemophilia Treatment (Sobi):

Swedish Orphan Biovitrum AB (Sobi) has received a favorable CHMP opinion for efanesoctocog Alfa, a novel once-weekly treatment for hemophilia.

Haemophilia A, a rare genetic condition, results from insufficient or dysfunctional factor VIII production, a crucial protein for blood clotting. Occurring in approximately one in 5,000 male births annually, it is less common in females. Individuals with haemophilia face bleeding episodes that can lead to joint damage, pain, and life-threatening hemorrhages. While treatment advancements have improved clinical outcomes, significant unmet clinical and social needs persist among those affected.

The recommendation from the CHMP stems from pivotal phase 3 studies: XTEND-1 for adults and adolescents and XTEND-Kids for children. These studies assessed the efficacy and safety of efanesoctocog alfa, demonstrating that weekly prophylaxis with efanesoctocog alfa (at 50 IU/kg) significantly reduced bleeds (mean ABR <1), with 80-88% of patients experiencing freedom from spontaneous bleeds, irrespective of age. No factor VIII inhibitors were detected in the efanesoctocog alfa clinical program.

Efanesoctocog alfa obtained its initial approval in the US from the Food and Drug Administration (FDA) in February 2023. Earlier, in May 2022, it received Breakthrough Therapy designation from the FDA, making it the first factor VIII therapy to earn this distinction. Additionally, it was granted Fast Track designation in February 2021 and Orphan Drug designation in 2017.

Opdivo (nivolumab) Combination Therapy for Urothelial Carcinoma (Bristol Myers Squibb):

Bristol Myers Squibb (BMS) has achieved a significant milestone with the CHMP's positive opinion regarding Opdivo (nivolumab) in combination with cisplatin and gemcitabine for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma.

The positive opinion from the CHMP is grounded in findings from a sub-study of the CheckMate -901 trial, presented at the European Society of Medical Oncology (ESMO) Congress 2023. In this sub-study, the combination of Opdivo with cisplatin and gemcitabine, followed by Opdivo monotherapy, exhibited statistically significant and clinically meaningful enhancements in primary efficacy measures: overall survival (OS) and progression-free survival (PFS), as evaluated by Blinded Independent Central Review (BICR).

With a median follow-up period of about 33 months, treatment involving Opdivo in conjunction with cisplatin and gemcitabine reduced the risk of death by 22%, demonstrating a median OS of 21.7 months versus 18.9 months observed with cisplatin-gemcitabine alone. Furthermore, patients receiving Opdivo combined with cisplatin and gemcitabine experienced a 28% reduction in the risk of disease progression or death, showcasing a median PFS of 7.9 months compared to 7.6 months with cisplatin-gemcitabine alone. Importantly, the safety profile remained consistent with the known safety profiles of the individual components of the regimen, and no new safety concerns emerged.

Fruzaqla (Fruquintinib) for Metastatic Colorectal Cancer (Takeda Pharmaceutical Company Limited):

Takeda Pharmaceutical Company Limited has received a positive CHMP opinion for Fruquintinib, an oral inhibitor of vascular endothelial growth factor receptor (VEGFR), intended for the treatment of metastatic colorectal cancer. This medication represents a significant advancement in the fight against colorectal cancer and offers a targeted approach to inhibiting disease progression.

Obgemsa (vibegron):

Obgemsa, offered in the form of 75 mg film-coated tablets, features vibegron as its active ingredient. Functioning as a urological medication, vibegron targets urinary frequency and incontinence, categorized under the ATC code G04BD15. Acting as a selective human beta 3-adrenoceptor (beta 3-AR) agonist, vibegron triggers the activation of the beta 3-AR in the bladder, leading to the flattening and elongation of the bladder base, thus facilitating urine storage.

The advantages of Obgemsa lie in its capacity to diminish the frequency of daily urination and incontinence episodes in patients with overactive bladder syndrome after a 12-week treatment period, when compared to a placebo. Furthermore, the effects of Obgemsa persist even after 52 weeks of continuous treatment.

Among the commonly reported side effects associated with Obgemsa are headache, diarrhea, nausea, constipation, urinary tract infection, and an increase in residual urine volume (the amount of urine left in the bladder after voluntary urination).

Obgemsa is specifically indicated for the symptomatic treatment of adult patients experiencing overactive bladder (OAB) syndrome.

Truqap (capivasertib):

Truqap, when used alongside fulvestrant, offers a significant benefit in terms of progression-free survival (PFS) for patients diagnosed with ER-positive, HER2-negative advanced breast cancer. Specifically, this benefit is observed in individuals who have experienced recurrence or progression after undergoing endocrine-based therapy and possess one or more PIK3CA/AKT1/PTEN alterations. When compared to a treatment regimen consisting of fulvestrant and placebo, Truqap demonstrates an enhanced PFS in these patient populations.

Among the most commonly reported side effects associated with Truqap are diarrhea, rash, nausea, fatigue, vomiting, stomatitis, hyperglycemia, headache, and decreased appetite.

Truqap is specifically indicated for the treatment of adult patients diagnosed with estrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer, who have one or more PIK3CA/AKT1/PTEN alterations, and have experienced recurrence or progression following prior endocrine-based therapy.

The CHMP has issued positive opinions on Tofidence (tocilizumab) and Wezenla (ustekinumab) during its April 2024 meeting.

Tofidence is intended for the treatment of rheumatoid arthritis, COVID-19, polyarticular juvenile idiopathic arthritis, and systemic juvenile idiopathic arthritis. Wezenla, on the other hand, is indicated for the treatment of plaque psoriasis, including pediatric plaque psoriasis, psoriatic arthritis, and Crohn’s disease.


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