USA Food and Drug Agency (FDA) approved Trikafta (elexacaftor / ivacaftor / tezacaftor), a first triple-combination therapy, which will be available for treatment of patients with the most common mutation in cystic fibrosis (CF).

Trikafta was approved for patients at 12 years of age, or more, who have at least one copy of F508del mutation in cystic fibrosis transmembrane conductance regulator (CFTR)gene, which corresponds to 90% of population with CF.

As stated by Norman Sharpless, FDA acting commissioner – “This decision allows for a new method for treatment of patients, including adolescents, and provides access to additional effective therapy”. He also added, that in the recent years, they have been witnessing a significant progress in treatment of CF, and an improvement in quality of life of patients, however many patient groups did not have approved treatment options. This was the reason behind using all available programs, to maximize the rate for approval of the treatment, while maintaining high treatment standards.

The effectiveness of Trikafta in patients with CF at 12 years of age or older, was demonstrated in two studies. The first study was a 24-week randomized double-blinded placebo-controlled trial, with 403 patients with, F508del mutation. Second study was a 4-week randomized double-blinded study with an active control, in 107 patients, with two identical F508delmutations.

The information for indication of Trikafta includes warnings, associated with risks of abnormal liver function (transaminases, and bilirubin), parallel use with other products, which act as inducers or inhibitors of another hepatic enzyme, cytochrome CYP3A, and cataract development.

Patients and healthcare personnel must discuss all details of treatment, prior to its administration.

Trikafta approval was handled by Vertex Pharmaceuticals Incorporated, which will also be receiving and additional voucher for acceleration of therapy development.

Link: gmpnews.ru

Residents of the MSU research park developed a unique treatment for battling sepsis. It has successfully passed all clinical trials and is soon expected to be available for use in hospitals. The new treatment is based on polymer sorbents derived for the first time.

The press office of the university reported that scientists have developed a novel method for deriving porous polymers of irregular structure, aimed at removing blood lipopolysaccharides, which are responsible for development of this malignant process.

“The structure is the following: porous spherical microgranules of hypercrosslinked styrene-divinylbenzene copolymer is used as a matrix. The surface of pores of microgranules has a covalently immobilized synthetic ligand to “Lipid A”, which is the most conserved domain of bacterial lipopolysaccharide. The proposed materials were chosen on the basis of safety, resistance to sterilization, absence of emission of low molecular weight compounds. Such matrix is characterized by high hemocompatibility, optimal morphology of pores (high proportion of mesopores to volume), and easiness of surface modification. Meanwhile ligand is characterized by strength of a bond to a lipopolysaccharide molecule” commented one of the developers, Ivan Bessonov.

Thуmedical device is a cylindrical plastic case, filled with the sorbent, that has standard ports for connection with blood tubing sets (“adsorber”, “adsorption column”).

With the help of a specialized pump, patient’s blood under a low pressure is filtered through the sorbent, while the blood cells and large plasma proteins are able to pass through without retention. Meanwhile, lipopolysaccharides are bound to specialized segments of sorbent surface.

Thereby, the purified blood is returned to the circulatory system, while the toxic substances, tightly bound by the sorbent, are removed from the organism.

The press office stated that thus far the production of the polymer sorbent and other component parts for manufacturing the adsorbers has been initialized. Independent accredited laboratories have conducted technical and toxicity experiment both in vivo and in vitroto test for biocompatibility and absence of toxic substances in materials that are utilized, and the efficiency of lipopolysaccharide adsorption. Poszdravnadzor conducted an expertise of experiment protocols and technological documentation, and allowed their use in clinical practice, recognizing them as safe and corresponding to the claimed characteristics.

The effectiveness of this extracorporeal blood filtration (selective hemosorption of lipopolysaccharides) is further confirmed by its recent inclusion in a pricelist of compulsory medical insurance in Moscow, Saint-Petersburg, and other regions.

“This method has already become a part of an insured medicine system. The company’s upcoming plans include new developments in the area of sorption technologies for blood filtration, and new products based on it, as well as extended (post registration) clinical trials on already existing products” commented the press office.

Link: gmpnews.ru

Takeda Russia announced the launch of two new indications of Adsetris® on the Russian market:

1) the treatment of previously untreated patients with classical CD30 + Hodgkin’s lymphoma stage IV in combination with doxorubicin, vinblastine and dacarbazine;
2) the treatment of patients with CD30 + T-cell lymphoma of the skin after at least one line of previous systemic therapy.

The targeted drug brentuximab vedotin (BV) is a CD30 conjugate of a directional monoclonal antibody and an antitumor agent monomethylauristatin E (MMAE).  Brentuximab vedotin binds to the CD30 receptor on the surface of the tumor cell and, penetrating inside, causes its apoptosis and death.

Adsetris® is available in the form of a lyophilisate for the preparation of a concentrate for the preparation of a solution for intravenous infusions.  BV first was registered in Russia on February 26, 2016.  for the treatment of patients with recurrent / refractory CD30 + Hodgkin lymphoma after autologous stem cell transplantation and patients with recurrent / refractory systemic anaplastic large cell lymphoma, and since December 26, 2016 it has also been registered for the treatment of patients with CD30 + Hodgkin lymphoma with an increased risk of recurrence or progression of the disease after autologous stem cell transplantation.

The goal of Takeda Company is to contribute to improving the quality of life of patients with the help of the latest achievements in medicine.

Registration on the Russian market of new testimony Adcetris® is a vivid example of the implementation of the course adopted by the company.
This antibody and its introduction into clinical practice provides patients who have exhausted the resources of classical treatment regimens, long-term remission, the possibility of carrying out transplantation of hematopoietic stem cells and gives them a chance for a complete cure.

Hodgkin’s lymphoma (HL) is a malignant tumor disease of the lymphatic system. HL are ill mainly at the age of 18-35 years.  In 95% of all cases, there is a CD30 antigen on the surface of HL cells, which is a therapeutic target for BV.

The main task of the first line of therapy for patients with HL is to achieve the maximum number of complete and long-term remissions.  Polychemotherapy (PCT) is the standard of treatment for HL in Russia, the USA and Europe, but despite this, 15-30% of patients who received first-line drugs develop relapses of the disease or primary resistance is observed.

The standard component of PCT, bleomycin, leads to pulmonary toxicity in 10% of patients, andmortality among patients with pulmonary toxicity due to bleomycin is observed in 10-20% of patients.
Thus, patients with newly diagnosed HL need more effective and safe therapy.

The results of BV are based on data from a Phase 3 registration study, ECHELON-1, in which patients with previously untreated classical Hodgkin’s lymphoma stage III or IV were included;  664 patients were assigned to the brentuximab group of vedotin, doxorubicin, vinblastine and dacarbazine (A + AVD) and 670 patients to the group of doxorubicin, bleomycin, vinblastine and dacarbazine (ABVD) [3]. The primary endpoint in the study adopted modified progression-free survival (mVBP).

Two-year MBHD in groups A + AVD and ABVD was 82.1% and 77.2% (p = 0.04).  The use of the A + AVD regimen by 23% reduced the risk of progression, death or the need for second-line therapy, which reduced the need for high-dose chemotherapy with stem cell autotransplantation by 33%.

The results of ECHELON-1 made it possible to register BV in combination with AVD for the treatment of patients with newly diagnosed HL in the USA and in Europe.

Link: www.clinvest.ru

Chinese scientists have begun the second phase of clinical trials of the drug for the treatment of an autoimmune disease – systemic lupus erythematous.

The new patent-protected drug SM934, which is a water-soluble artemisinin derivative, was developed by researchers at the Shanghai Institute of Pharmacology at the Academy of Sciences of China ANC.

The first stage of clinical trials of a new drug has already been completed, and the second stage began in one of the Shanghai hospitals.

Systemic lupus erythematous, one of the most common types of lupus erythematous, is an autoimmune disease in which the human immune system mistakenly attacks healthy tissues of the body, causing damage to the joints, skin, kidneys, blood cells, brain, heart and lungs.

One of the hallmarks of lupus is a rash on the face, resembling butterfly wings in outline.
In China, nearly one million people are reported to have lupus erythematous.

Since the causes of the origin and development of the disease remain unclear, the development of new drugs for this disease causes difficulties.

According to the ANC, the drug SM934 is an oral drug with a low dosage and a pronounced effect.  It can modulate autoimmune reactions and restore the body’s immune balance.

Link: gmpnews.ru