New Brain-Administered Gene Therapy; New drugs for: UTIs, Hemophilia, Stomach Cancer, Breast Cancer
Gene Therapy Kebilidi Stimulates Brain’s Dopamine Production
The U.S. Food and Drug Administration (FDA) granted “accelerated approval” for PTC Therapeutics’ Kebilidi, the first gene therapy treating a rare inherited neurological disorder called AADC (aromatic L-amino acid decarboxylase) deficiency. Kebilidi is also the first gene therapy that is inserted directly into the brain. Accelerated approval status means that full approval is contingent on clinical studies results.
In its press release, PTC Therapeutics explains that “AADC deficiency is a fatal, rare genetic disorder that typically causes severe disability and suffering from the first months of life, affecting every aspect of life—physical, mental and behavioral.” The lack of the enzyme AADC reduces the ability of the brain to synthesize dopamine, a neurotransmitter essential for motor function. Children with the disorder experience delays in motor control function (head control, sitting, standing, and walking), hypotonia (weak muscle tone), and developmental and cognitive delays.
Kebilidi delivers a functioning human DDC gene directly into the brain in order to boost the amount of AADC and restore dopamine production.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-aromatic-l-amino-acid-decarboxylase-deficiency
More on this approval: https://www.fiercepharma.com/pharma/first-brain-delivered-gene-therapy-hits-us-ptc-therapeutics-aadc-deficiency-treatment
Read a case study of 16-month-old boy who became the youngest child in the world to receive the experimental one-time gene therapy for AADC deficiency. The treatment, now known as Kebilidi, was administered at Cincinnati Children’s Hospital in November 2022. The complicated procedure was succesful and the child’s progress has been remarkable. This report includes photos before and after the treatment.
https://scienceblog.cincinnatichildrens.org/the-joy-of-witnessing-science-transform-a-life/
Uncomplicated UTIs: Orlynvah
The FDA approved Iterum Therapeutics' Orlynvah tablets to treat uncomplicated urinary tract infections caused by Escherichia coli, Klebsiella pneumoniae, or Proteus mirabilis in adult women who have limited or no alternative oral antibacterial treatment options (due to an allergy or antibiotic resistance). In clinical trials, the most common side effects of Orlynvah were diarrhea, nausea, vaginal yeast infection, headache, and vomiting.
https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-new-treatment-uncomplicated-urinary-tract-infections-adult-women-who-have-limited-or-no
More on this approval: https://gizmodo.com/new-antibiotic-hits-the-market-to-tackle-stubborn-utis-2000517862
Hemophilia: Hympavzi
The FDA approved Pfizer’s Hympavzi (marstacimab-hncq) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A (more common) or B. Patients with hemophilia are unable to clot properly and may bleed for a longer time than normal after injury or surgery. They may also have spontaneous bleeding in muscles, joints and organs, which can be life-threatening. The most common types of hemophilia are inherited (on the X chromosome), and occurs in about 1 of every 5,000 male births.
https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-hemophilia-or-b
Read more about hemophilia, including diagnosis and treatment, at the Centers for Disease Control: https://www.cdc.gov/hemophilia/about/index.html
Stomach Cancer: Vyloy
The FDA approved Astellas Pharma's Vyloy (zolbetuximab-clzb), to be used in conjunction with chemotherapy, for treating advanced stomach cancer or gastroesophageal junction (GEJ) cancer that’s positive a certain type of protein that promotes tumor growth and spread. It also approved a companion diagnostic test to identify patients who may be eligible for treatment with zolbetuximab.
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zolbetuximab-clzb-chemotherapy-gastric-or-gastroesophageal-junction-adenocarcinoma
Breast Cancer: Itoveb
Roche’s Itovebi (inavolisib) received FDA approval for the treatment of certain kind of breast cancer with a PIK3CA mutation. It is to be used in combination with two other drugs (Ibrance and fulvestrant).
https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-inavolisib-palbociclib-and-fulvestrant-endocrine-resistant-pik3ca-mutated-hr-positive
Check out November's recommended articles and podcasts here:
The November 2024 Drug Safety Update Part 3 will be sent later today.