Editors' (re)View: A busy CGT week; Old therapy, new tricks

June 23, 2023
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of June 19.

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.

—Karen Langhauser

About the Author

Andrea Corona | Senior Editor

Andrea Corona serves as the Senior Editor of Pharma Manufacturing — a leading source of news and insights for pharma professionals — and is responsible for creation of editorial content, moderating webinars, and co-hosting the "Off script" podcast. Her editorial journey started as an as associate editor at Biocompare, an online platform providing product information, industry news, articles, and other resources to support scientists in their work. Before Biocompare, she was a digital producer at Science Friday, focusing on adapting radio segments for the web and social media management. Andrea earned her bachelor's degree in journalism and biology from the State University of New York, at Purchase College. 
About the Author

Karen P. Langhauser | Chief Content Director, Pharma Manufacturing

Karen currently serves as Pharma Manufacturing's chief content director.

Now having dedicated her entire career to b2b journalism, Karen got her start writing for Food Manufacturing magazine. She made the decision to trade food for drugs in 2013, when she joined Putman Media as the digital content manager for Pharma Manufacturing, later taking the helm on the brand in 2016.

As an award-winning journalist with 20+ years experience writing in the manufacturing space, Karen passionately believes that b2b content does not have to suck. As the content director, her ongoing mission has been to keep Pharma Manufacturing's editorial look, tone and content fresh and accessible.

Karen graduated with honors from Bucknell University, where she majored in English and played Division 1 softball for the Bison. Happily living in NJ's famed Asbury Park, Karen is a retired Garden State Rollergirl, known to the roller derby community as the 'Predator-in-Chief.'