Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.
A busy CGT week
This week, we reported on two important milestones in the ongoing journey to harness the power of gene therapy and bring life-changing treatments to those affected by rare genetic diseases.
Sarepta Therapeutics announced today that it has obtained accelerated approval from the FDA for its gene therapy, Elevidys. This adeno-associated virus-based therapy has been authorized for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene. The approval of Elevidys marks a significant milestone in Duchenne treatment, being the first and only gene therapy approved for this degenerative disease. Sarepta's CEO, Douglas Ingram, emphasized the therapy's potential to alter the course of Duchenne and acknowledged the collaborative effort involved in achieving this milestone.
In another development, the FDA accepted bluebird bio's BLA for its sickle cell gene therapy, lovo-cel, with priority review. Lovo-cel aims to introduce a functional copy of the gene responsible for adult hemoglobin production, addressing the underlying cause of sickle cell disease. The BLA submission journey has had its challenges, but bluebird bio remains committed to advancing this one-time treatment option. If approved, lovo-cel would become bluebird bio's third ex-vivo gene therapy to receive FDA approval for a rare genetic disease. The therapy has a PDUFA goal date of December 20, 2023.
The approval of Elevidys represents a breakthrough for Duchenne treatment, offering hope to patients and their families by providing a potential treatment option that can alter the trajectory of this debilitating disease. It sets a precedent as the first and only gene therapy authorized for Duchenne, paving the way for further advancements in the field.
Similarly, the acceptance of lovo-cel's BLA for sickle cell disease underscores the potential of gene therapy to address the underlying cause of genetic disorders. If approved, lovo-cel would offer a one-time treatment approach that could significantly improve the lives of individuals living with sickle cell disease, a condition that has long lacked targeted therapies.
— Andrea Corona
Teaching an old therapy new tricks
This week, we reported that the FDA approved Agepha Pharma's Lodoco (colchicine) tablets — marking the first approved drug to target cardiovascular inflammation and the Slovakia-based company’s first product launch in the U.S.
But it wasn't a first for colchicine. Colchicine is a plant-based alkaloid extracted from autumn crocus, meadow saffron and glory lilies. Its medicinal use dates back to ancient Egypt, where manuscripts noted its use as an herbal remedy for joint pain.
The FDA licensed it back in 1961, but this was before the modern day drug approval system was in place. In October 1962, Congress passed the Kefauver-Harris Drug Amendments to the Federal FD&C Act which meant that before marketing a drug, companies had to prove not only safety, but effectiveness.
Colchicine was then formally approved under the Unapproved Drug Initiative in 2009 for familial mediterranean fever — an autoinflammatory genetic disorder. It lost patent protection in 2014.
According to Agepha, Lodoco has been "reformulated specifically for long-term use in cardiovascular disease patients and there are no generic alternatives.”
Coronary artery disease (CAD) — a type of heart disease — is a leading cause of death in the U.S. Both inflammation and high cholesterol have been shown to contribute to cardiovascular risk. Statin therapies are commonly used to lower cholesterol but patients still struggle with residual inflammation. Enter colchicine, which has been shown to suppress local cardiac production of inflammatory cytokines IL-1β, IL18, and IL-6 in patients with CAD.
In fact, according to Agepha's studies, Lodoco can reduce the risk of cardiac events in patients with established cardiovascular diseases by 31% on top of standard of care. Patients already taking a statin can add the anti-inflammatory drug to their daily routine and lower their risks of heart attack and stroke — a welcome new trick for a 5,000-year old medicine.