Pfizer and OPKO Health: A step closer to FDA Nod for pediatric growth hormone deficiency (GHD) drug, Somatrogon
Pfizer and Opko had joined hands in 2014 to develop somatrogon, a pediatric growth hormone deficiency (GHD) drug. It comprises the natural sequence of growth hormone and a copy of the C-terminal peptide (CTP) from the beta chain of human chorionic hCG at the N-terminus and two copies at the C-terminus, which helps in extending the half-life of the molecule.
The drug had already been rewarded with Orphan Drug designation by the US FDA and EU for the treatment of children and adults with growth hormone deficiency. Growth hormone deficiency (GHD) is a condition that arises due to the lack of enough growth hormone in the body. It can be due to a damaged pituitary gland or another gland called the hypothalamus.
Recent results from Phase III clinical studies support the duo’s regulatory submission for somatrogon. The US FDA has accepted the initial Biologics License Application submission for the therapy with the target action date for an FDA decision is October 2021. Somatrogon is going to compete with already established Pfizer’s Genotropin, a recombinant human growth hormone, however, the latter one puts a burden on the patients due to its daily injections, which also leads to compliance issues in children.
FDA Emergency Use Authorization for SCONE Medical’s Novel Aerosol Infection Containment Device
SCONE Medical Solutions Inc. (SMS) has collaborated with Mayo Clinic to fight the battle against infectious diseases. The idea is to provide protection to healthcare workers taking care of infected patients during Aerosol Generating Procedures (AGPs), particularly in the acute care/triage setting.
As a pre-gift of the new year, the companies received the Emergency Use Authorization from the FDA for the SCONE device. As the whole world is busy fighting the infectious disease that keeps on re emerging and mutating, SCONE is building a future that is safer due to its effective substitute to manage the aerosol transfer among different patients and employees.
The doublet is working together on developing SCONE, a small capacity, disposable device. It leverages the negative pressure to vacuum out aerosols emitted around a patient’s head and neck, thereby cushioning with an extra layer of “active” barrier protection for healthcare workers while treating potentially infectious patients. Easily portable and disposable, the device provides an added advantage.
Antengene Files for NDA for ATG-010 (Selinexor) in South Korea for rrMM and rrDLBCL
Antengene Corporation has submitted a New Drug Application (NDA) with Orphan Drug Designation (ODD) to the South Korean Ministry of Food and Drug Safety (MFDS) for ATG-010 (selinexor, XPOVIO) in combination with low dose dexamethasone for the treatment of adult patients with relapsed/refractory multiple myeloma (rrMM).
Furthermore, the company has also gone forward for the filing for the NDA of ATG-010 as monotherapy to treat adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL).
The submissions came after the positive results of the pivotal STORM and SADAL studies that demonstrated significant and meaningful efficacy with a manageable safety profile for ATG-010. ATG-010 is a first-in-class and only-in-class oral selective inhibitor of nuclear export (SINE). It is the first FDA-approved to be used for the treatment of both multiple myeloma and diffuse large B-cell lymphoma.
Currently, Antengene is conducting two registrational Phase 2 clinical trials of ATG-010 in China for rrMM (MARCH) and for rrDLBCL (SEARCH). The drug has already received orphan drug designation in South Korea in October 2020. Its US partner, Karyopharm received FDA approval for the treatment of patients with multiple myeloma after at least one prior therapy on December 18, 2020.
SanBio Publishes the Results of STEMTRA Phase 2 Interim Analysis for SB623 in Neurology
SanBio Group recently publicized the interim analysis of SB623 Phase 2 clinical “STEM cell therapy for TRAumatic brain injury” (STEMTRA) trial in an online issue of Neurology, the medical journal of the American Academy of Neurology.
STEMTRA was a Phase 2, randomized, double-blind, surgical sham-controlled, global trial aimed at investigating the efficacy and safety of SB623 compared to sham surgery in patients with stable chronic neurological motor deficits secondary to traumatic brain injury (TBI).
The trial managed to achieve the primary efficacy endpoint of significant improvement from baseline of Fugl-Meyer Motor Scale (FMMS) score at six months for SB623-treated patients. While secondary endpoints did manage to improve from baseline but were not statistically significant versus control
The results of the STEMTRA trial have opened new doors in the chronic brain dysfunction domain, a relatively difficult area for drug development. However, several start-up companies have taken initiative to advance the therapeutic landscape in the field.