Published by the Spanish Agency for Medicines and Health Products (AEMPS) Ten new treatment positioning reports (IPT) Four oncology drugs and six more to treat cholestasis, pneumonia, alopecia, neurofibromatosis, diabetes and COVID-19. The purpose of these reports is to evaluate whether drugs for human use are effectively integrated into healthcare practice.
Oncology is the field with the most IPTs. Specifically, Spanish regulators issued a report on Abecma (vicleucel), a gene therapy drug containing T lymphocytes extracted from patients (autologous) for use in immunotherapy.It is suitable for adult patients Relapsed and refractory multiple myelomaReceived at least three prior therapies, including PI, IMD, and anti-CD38 mAb, and had disease progression on the last therapy. The treatment’s efficacy was demonstrated in the major Phase II trial BB2121-MM-001 (MM-001, KarMMa-1), an open-label, multicenter, single-arm (uncontrolled) trial in patients with RRMM Adult patients. Have received at least three prior therapies, including IMD, PI, and anti-CD38 mAb, and have been refractory to the last line of therapy.
Carvykti (ciltacabtagene autoleucel) This is another multiple myeloma drug that has received a report from AEMPS.This is another gene therapy for adult patients Relapsed and refractory multiple myelomaReceived at least three prior therapies, including PI, IMD, and anti-CD38 mAb, and had disease progression on the last therapy. The efficacy of cilta-cel was demonstrated in the pivotal MMY2001, CARTITUDE-1, Phase 1b/2, multicenter, single-arm, open-label trial evaluating the efficacy and safety of cilta-cel in adult patients with RRMM and safety. Have received at least three prior therapies, including PIs, IMD drugs, and anti-CD38 mAbs, and their disease has progressed during or within 12 months after the last line of therapy.
AEMPS re-releases IPT’s next oncology drug enkhtu (Trastuzumab (drutican)expressed as monotherapy gastric adenocarcinoma or patients with advanced HER2-positive gastroesophageal junction who have received a course of trastuzumab. Efficacy of T-DXd in the treatment of adult patients with advanced gastric ADC or HER2-positive GEJ (ADC G/HER2+ GEJ) who have received at least one prior course of trastuzumab based on final results from the pivotal study DS8201 – A-U205 (DESTINY Gastric02 ) and from supporting study DS8201-A-J202 (DESTINY Gastric01).
The latest published oncology drug report is Senblix (Assimini), Show that in chronic myelogenous leukemia In chronic phase and Philadelphia chromosome positive (CML-FC Ph+) who have been previously treated with two or more tyrosine kinase inhibitors. Efficacy data for aximinib in third-line Ph+ CML-CF are from a preliminary analysis of a phase 3 study (NCT03106779 or ASCEMBL) (ongoing) and from a phase 1 subgroup of CML-CF patients without the T315I mutation Supporting data for my study.
Cholestasis, pneumonia, alopecia areata, neurofibromatosis, diabetes and COVID-19
Outside of oncology, AEMPS releases another IPT Bylvay (orvisibate)suitable for treatment progressive familial intrahepatic cholestasis Patients six months or older. The efficacy of odevixibat has been evaluated in two clinical trials: A4250-005 (NCT03566238, EudraCT2017-002338-21), considered pivotal; Supportive open label extension phase.
for pneumoniaSpanish regulator issues another IPT Quofenix (derafloxacin), indicated for the treatment of acute bacterial infections of the skin and related skin structures (ABSSSI) and community-acquired pneumonia (CAP) in adults. The evidence for the efficacy of delafloxacin in the NAC indication was evaluated in a multicenter, double-blind, randomized, controlled Phase III clinical trial (ML-3341-306), which compared intravenous and oral delafloxacin with intravenous moxissa star, with the option to switch to oral administration, in 859 adults with CAP and Pneumonia Patient Outcomes Study Group risk categories II to V.
Olumiant (Baricitinib) has been the subject of another IPT. In this case, the treatment is as follows: severe alopecia areata in adult patients. The efficacy and safety of baricitinib were evaluated in two clinical trials: an adaptive Phase II/III trial (BRAVE-AA1, also known as I4V-MC-JAHO) and a third Phase 1 trial (BRAVE-AA2, also known as 14V-MC-JAL). Efficacy was demonstrated in Phase III of these studies, which were randomized, double-blind, parallel, placebo-controlled, lasting 36 weeks, with an extension phase of up to 200 weeks.
Koselugo (selumetinib)for patients aged 3 to 18 years with inoperable plexiform neurofibromas neurofibromatosis type 1, has become the subject of another IPT issued by AEMPS. Selumetinib efficacy data come from the SPRINT Phase I and SPRINT Phase II Stratum 1 studies.
Another published report is Mounjaro (tezepatide)a treatment for adult patients type 2 diabetes Insufficient controls related to diet and exercise. The efficacy of TZP has been evaluated in five pivotal randomized controlled phase 3 studies (SURPASS 1-5) and two Japanese population studies (GPGO and GPGP), and these studies were considered supportive. Two regional phase III studies (GPGO and GPGP) were considered supportive and were 52-week multicenter studies conducted in Japan. The GPGO study is a double-blind study designed to evaluate the safety and efficacy of TZP monotherapy compared with dulaglutide (DULA) 0.75 mg. The GPGP study was an open-label, noncomparative study designed to evaluate the safety and efficacy of TZP in combination with another oral antidiabetic drug (OAD).
Finally, the last IPT Ronapreve (casirivimab/imdevimab)indicating treatment and Prevent COVID-19 infection. The Adaptation Study of casirivimab/imdevimab in non-hospitalized patients evaluated the efficacy of casirivimab/imdevimab in patients with SARS-CoV-2 infection compared with placebo (COV-2067 trial) and using a sequential phase design: Phase I ( Tolerability and safety assessment compared to placebo), Phase II (viral load reduction assessment compared to placebo), and Phase III, in which three cohorts evaluated Cohort 1. It was this phase III that proposed the design for evaluating efficacy. Casirivimab/imdevimab versus placebo in preventing hospitalization and/or death.