JANUARY 2023 CHMP POSITIVE OPINION
BMS's Sotyktu received CHMP's positive opinion for psoriasis.
The Committee for Medicinal Products for Human Use (CHMP) recommended the approval of Sotyktu (deucravacitinib) for treating adult patients with moderate-to-severe plaque psoriasis.
The positive recommendation was based on the Phase 3 POETYK PSO-1 and POETYK PSO-2 trials, demonstrating the efficacy versus placebo and Otezla.
In the POETYK PSO-1 trial, the Psoriasis Area and Severity Index (PASI 75) and static Physician's Global Assessment score of 0 or 1 (sPGA 0/1) were the primary endpoints. In patients on Sotyktu, PASI75 at week 16 was 58.4% versus 35.1% in patients on Otezla. sPGA 0/1 was 53.6% in the Sotkyu arm versus 32.1% in patients on Otezla.
In the POETYK PSO-2 trial, PASI 75 was 53.0% on Sotkyu versus 39.8% on Otezla; sPGA 0/1 was 49.5% on Sotkyu versus 33.9% on patients on Otezla.
Sotyktu is the allosteric tyrosine kinase 2 (TYK2) inhibitor approved previously in the US for psoriasis. Sotkyu demonstrated superiority over Otezla and was well tolerated.
DECEMBER 2022 CHMP POSITIVE OPINIONS
CSL's gene therapy, Hemgenix, received a positive CHMP opinion for treating Hemophilia B.
The CHMP gave a positive opinion for Hemgenix (etranacogene dezaparvovec), a gene therapy for treating severe and moderately severe Haemophilia B.
The approval was based on the HOPE-B trial, demonstrating the efficacy of Hemgenix in patients with hemophilia B. Hemgenix showed a stable and durable increase in mean Factor IX activity, and the annual bleeding rate was reduced by 64%.
Haemophilia B is a rare disease resulting in bleeding in joints, muscles, and internal organs, leading to pain, swelling, and joint damage. The current standard of care is the infusion of factor IX for a lifetime.
Imjudo and Imfinzi combination received CHMP positive opinion for advanced liver and lung cancers.
AstraZeneca's Imfinzi (durvalumab) and Imjudo (tremelimumab) combination received CHMP positive opinion as first-line therapy for patients with advanced or unresectable hepatocellular carcinoma (HCC), and Imfinzi, Imjudo, chemotherapy combination in patients with Stave IV non-small cell lung cancer (NSCLC).
The approval as first-line treatment in liver cancer patients was based on the Phase 3 HIMALAYA trial. STRIDE regimen (Single Tremelimumab Regular Interval Durvalumab) reduced the risk of death by 22% versus sorafenib. The median OS was 16.4 months in patients on the STRIDE regimen versus 18.4 months in patients on sorafenib.
Positive opinion for Stage IV lung cancer s based on the POSEIDON Phase 3 trial. Anti-CTLA-4 antibody Imjudo, Imfinzi, and four cycles of platinum-based chemotherapy reduced the risk of death by 23% versus chemotherapy alone. After five years, 31% of patients on the Imjudo combination were alive versus 22% of patients on chemotherapy.
Pombiliti received CHMP positive opinion for Pompe disease.
Amicus Therapeutics' Pombiliti (cipaglucosidase alfa) received CHMP positive opinion for late-onset Pompe Disease.
The positive opinion was based on Phase 3 pivotal study (PROPEL) study, which recruited high unmet need enzyme replacement therapy (ERT)-experienced and ERT-naive patients.
NOVEMBER 2022 CHMP POSITIVE OPINIONS
GSK and Sanofi's VidPrevtyn received CHMP positive opinion as a booster dose to prevent COVID-19
GSK and Sanofi's VidPrevtyn Beta received CHMP positive opinion as a booster dose in adults previously administered with an mRNA or adenoviral vector COVID-19 vaccine.
Sanofi announced that it was the first protein-based adjuvanted COVID-19 vaccine approved in Europe. Further, the companies announced that the vaccine is effective against all variants of COVID-19.
The positive opinion was based on Phase 3 VAT08 Stage 2 trial, in which 23,726 individuals were recruited. Sanofi announced that the next-generation vaccine was more effective in stimulating immune response than Pfizer-BioNTech's COVID-19 vaccine.
Sanofi's Dupixent received CHMP positive opinion for prurigo nodularis
Dupixent (dupilumab) received CHMP positive opinion to treat adults with moderate-to-severe prurigo nodularis who are candidates for systemic therapy.
The CHMP positive opinion was based on two Phase 3 trials, PRIME and PRIME2; Dupixent reduced itching and skin lesions versus placebo. There was an improvement in health-related quality and a reduction in skin pain and symptoms of anxiety/depression.
Adverse events were higher in the Dupixent arm versus the placebo.
Enhertu received CHMP positive opinion for gastric cancer.
Daiichi Sankyo received CHMP positive opinion for Enhertu (trastuzumab deruxtecan) to treat adults with HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
Bayer's Eylea received CHMP positive for treating preterm infants with retinopathy of prematurity.
Eylea (aflibercept) received CHMP positive opinion for treating preterm infants with retinopathy of prematurity (ROP).
The CHMP positive opinion was based on Phase III study FIREFLEYE, in which Eylea failed to show non-inferiority versus laser photocoagulation (85.5% on intravitreal Eylea vs. 82.1% with laser photocoagulation).
Laser photocoagulation was associated with the destruction of retinal tissue, which might lead to high myopia (nearsightedness) and peripheral vision loss.
With the indication extension, Bayer aims to expand the patent protection period of Eylea by six months. Bayer seeks to extend the patent until November 2025 with the patent extension.
AstraZeneca's Imfinzi received a positive CHMP opinion for treating biliary tract cancer.
Imfinzi received CHMP positive opinion for treating adults with unresectable or metastatic biliary tract cancer. Imfinzi has to administer in combination with gemcitabine or cisplatin.
OCTOBER 2022 CHMP POSITIVE OPINIONS
Takeda's Dengue Tetravalent Vaccine received CHMP positive opinion.
Takeda's Dengue vaccine received CHMP positive opinion and is active against genotypes 1, 2, 3, and 4. The vaccine received CHMP positive opinion for four years and older.
Nearly 20,000 to 25,000 per year die because of dengue infection, primarily children. The vaccine protects against fever, hospitalization, and death. The vaccine also received CHMP positive opinion under the brand name Qdenga.
The first therapy for the post-transplant lymphoproliferative disease, Ebvallo, received positive CHMP opinion.
Atara Biotherapeutics and Pierre Fabre's Ebvallo (tabelecleucel) received CHMP positive opinion for Epstein-Barr virus-positive post-transplant lymphoproliferative disease. Ebvallo received a positive opinion for treating adults and pediatric patients two years and older.
Ebvallo is Epstein-Barr virus (EBV)-specific T-cell immunotherapy developed for EBV-driven diseases, including post-transplant lymphoproliferative disease (EBV + PTLD).
The approval was based on the Phase 3 ALLELE Study, demonstrating a favorable risk-benefit profile. The median overall survival was nearly 1.7 months in EBV + PTLD following hematopoietic cell transplant (HCT) and almost three months in solid organ transplant (SOT) recipients with EBV + PTLD. SOT and HCT patients received a median of two and three cycles. The objective response rate was 50% in the overall population and 50% each in SOT and HCT patients. The median overall survival was 18.4 months.
Novartis' Pluvicto received CHMP positive opinion for PSMA-positive metastatic castration-resistant prostate cancer.
CHMP has given a positive opinion for Pluvicto (lutetium (177Lu) vipivotide tetraxetan) to treat adult patients with PSMA-positive metastatic castration-resistant prostate cancer.
The CHMP positive opinion was based on the Phase III VISION study, in which Pluvicto plus standard of care showed improvement in overall survival versus standard of care alone in patients previously treated with AR pathway and taxane-based chemotherapy. The risk of death was reduced by 38%, and the risk of radiographic progression or death was reduced by 60% in patients on Pluvicto plus standard of care versus standard of care alone.
If approved, Pluvicto will be the first radioligand therapy available for prostate cancer in Europe.
After FDA approval, BI's Spevigo received CHMP positive opinion for generalized pustular psoriasis.
Boehringer Ingelheim's Spevigo (spesolimab) received CHMP positive opinion for patients with generalized pustular psoriasis (GPP) flares.
The approval was based on EFFISAYIL 1 trial, a 12-week Phase 2 trial in patients with GPP flares. 900 mg of IV spesolimab was tested versus placebo. One week after treatment with spesolimab, 54% of patients showed no visible pustules versus 6% of patients on placebo. After 12 weeks of treatment, 84.4% of patients had no visible pustules and almost had clear skin.
Spesolimab is an interleukin-36 receptor (IL-36R) inhibitor that received approval in the US and Japan.
BeiGene's Brukinsa received CHMP positive opinion for adults with CLL
BeiGene's Brukinsa (zanubrutinib) received a positive CHMP opinion for treating adult patients with chronic lymphocytic leukemia (CLL).
The CHMP positive opinion was based on a global head-to-head Phase 3 clinical study, ALPINE trial. Brukinsa (zanubrutinib) was tested versus ibrutinib in patients with relapsed or refractory chronic lymphocytic leukemia. In the SEQUOIA trial, Brukinsa was tested versus bendamustine plus rituximab in patients with previously untreated CLL or SLL.
Libtayo received CHMP positive opinion for cervical cancer
Libtayo (cemiplimab) received CHMP positive opinion for treating advanced cervical cancer. The recommendation was based on the Phase 3 trial, demonstrating significant improvement in survival versus chemotherapy, irrespective of PD-L1 status.
The approval was based on the Phase 3 EMPOWER-Cervical 1 trial, which recruited 608 patients. The primary endpoint was overall survival, which reduced the risk of death by 31% and 27% in squamous cell carcinoma.
Libtayo was approved for basal cell carcinoma, cutaneous squamous cell carcinoma (CSCC), and advanced non-small cell lung cancer (NSCLC).
SEPTEMBER 2022 CHMP POSITIVE OPINIONS
Beyfortus (nirsevimab) for preventing infection of respiratory syncytial virus (RSV)
CHMP gave a positive opinion for AstraZeneca's Beyfortus (nirsevimab) to prevent respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants during their first RSV season.
The positive opinion was based on MELODY Phase III, MEDLEY Phase II/III, and Phase IIb trials. In the studies, Beyfortus met the primary endpoint of reduction in the medically attending lower respiratory tract infection incidence versus placebo when administered as a single dose. There were no major differences in the safety outcomes between Beyfortus and placebo.
RSV infections are the most common respiratory infections leading to the hospitalization of infants. The current standard of care options is limited to treatments that provide symptomatic relief.
Enjaymo (sutimlimab) for hemolytic anemia in adult patients with cold agglutinin disease
Genzyme's Enjaymo (sutimlimab) received a positive CHMP opinion for treating hemolytic anemia in adult patients with cold agglutinin.