Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.
(Less) heavy lies the crown...will there be a new king in town?
Earlier this week, Eli Lilly announced a successful phase 3 trial evaluating tirzepatide for managing chronic weight issues. The trial, which involved 938 adult participants who had type 2 diabetes and were either overweight or obese, met all primary and secondary objectives.
Based on these results, Lilly plans to complete its FDA submission for tirzepatide in obese or overweight adults in the coming weeks and expects regulatory action as early as the end of the year. Given that the drug was already approved by the FDA last May to improve glycemic control in adults with type 2 diabetes, it will likely get the green light from the regulator.
The huge news here is that analysts are predicting tirzepatide has the potential to usurp Humira to become the best-selling drug of all time. Millions of patients have been treated with Humira worldwide, and it’s estimated that it will amass a revenue of $240 billion by 2024. Some analysts are predicting that tirzepatide could generate annual sales of $25 billion — a figure that would exceed the previous record of $20.7 billion set by AbbVie's Humira in 2021.
A few months ago, Ozempic, a drug approved to treat diabetes, dominated headlines as celebrities and internet influencers sang its praises when used off-label as a weight loss drug. (On TikTok, the hashtag #Ozempic has been viewed over 888 million times.)
The demand, coupled with some CMO-related manufacturing snags, has caused a shortage of both Novo Nordisk's semaglutides — Ozempic and the version actually approved for weight loss, Wegovy.
This momentum, coupled with a large potential market — Novo estimates that there are 764 million people currently living with obesity globally — will most likely capture the pharma industry's attention.
While treatments are currently cost-prohibitive (and typically not covered by insurers), companies are working to get costs down by formulating oral pills (Novo has a long-acting, oral GLP-1 analogue intended for once-daily treatment in phase 3 trials). If the buzz continues, the sector might just crown the next king (or queen) of blockbusters...and they may be sleeker than ever.
First-in-class ALS treatment brings hope
On Wednesday, we reported that the FDA has given its approval to Biogen's Qalsody, a drug designed to treat a rare and uniformly fatal form of amyotrophic lateral sclerosis (ALS) caused by a mutation in the superoxide dismutase 1 (SOD1) gene.
Even though the ultra-rare mutation only affects around 330 people in the US, the drug brings a glimmer of hope as the first approved treatment to target a genetic cause of ALS. The drug is an antisense oligonucleotide that works by blocking the production of the SOD1 protein, which is known to clump together and damage the nervous system.
It's been a promising year for ALS treatments. Back in September Amylyx scored FDA approval for its broader ALS drug, now branded as Relyvrio, an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol, designed to preserve neurons by blocking cell death pathways in the mitochondria and endoplasmic reticulum. Biogen has said that Qalsody will be priced similarly to other approved ALS treatments like Relyvrio, which will have a list price of $160,000 per year.
According to Biogen's CEO Chris Viehbacher, the agency's nod signifies a “consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS.” Jean Swidler, chair of Genetic ALS & FTD: End the Legacy, echoed his excitement saying, "We are excited to see what future therapies are developed now that it is understood that lowering levels of neurofilament provides important evidence that a treatment is affecting the neurodegenerative process.”
Neurofilaments are structural proteins found in neurons, and its levels can increase in cerebrospinal fluid and blood when there is neuronal damage or degeneration. The protein has been evaluated as a biomarker for other central nervous diseases such as multiple sclerosis and Parkinson's.
While Biogen's approval is still contingent on confirmatory data, Qalsody could change the game for neurofilament targeting drugs.