Therapeutic areas in focus
Furosemide approved by FDA for patients with chronic heart failure
The FDA has approved furosemide (Furoscix) injections as an at-home treatment for patients with chronic heart failure, suffering from congestion.
It is administered through a system known as an On-Body Infuser.
According to scPharmaceuticals, heart failure affects more than six million people in the US and costs over $30bn annually, so another treatment option is hugely positive news. The new treatment is approved for treating congestion caused by fluid overload in patients with chronic heart failure who do not need hospitalisation, but have not responded to oral diuretic treatments.
Furoscix lowers fluid overload in patients to treat their congestion, and it is the only self-administered loop diuretic currently on the market. It is administered subcutaneously using a wearable, pre-programmed delivery device.
In the phase 2 trial, 51 patients were recruited, and the new treatment was compared with standard care over a 30-day period. Those treated with furosemide showed a 37% lower risk of hospitalisation from heart failure when compared to those treated with the traditional method.
John Tucker, President and Chief Executive Officer of scPharmaceuticals, said, “Congestion due to worsening heart failure is one of the most common causes of hospital admissions in patients over 65, and today’s approval of Furoscix represents an important treatment advancement for the over 7 million heart failure patients in the US that will be able to self-administer IV equivalent diuresis at home. […] We are preparing to optimise commercialisation efforts to offer Furoscix to patients in the first quarter of next year with the goal of driving rapid patient adoption to meet the needs of the $5.9bn addressable market in the US.”
Controversial ALS drug approved by FDA
Although scientists and independent medical experts questioned the drug’s efficacy, the FDA has approved a new drug called Relyviro to treat patients with amyotrophic lateral sclerosis (ALS). The drug has been shown to slow symptoms and extend life, albeit only by a few months.
Most patients with ALS die within three to five years and multiple trials for various ALS drugs and treatments have failed in recent years, so the development and approval of a drug that can extend life by even a few months can only be a positive step for those suffering from ALS.
Initially, the advisory panel recommended that the FDA reject the drug, arguing that Amylyx hadn’t made a convincing case for the efficacy of the drug. However, after pleas from patients and their families, the FDA reconvened the panel this month. More data and further analyses were presented, suggesting that the drug will allow patients with ALS to live for up to 11 months longer than previously expected.
Only two drugs treating ALS have been approved since 1995, and neither is particularly effective, so this controversial approval is a significant step in the treatment of ALS.
Despite the uncertainty around the drug and the controversy of its approval, patients with ALS are eager to use the drug. Marc Litt, a retired cardiologist who was diagnosed with ALS in 2019, states: “Obviously, I realise any treatment won’t cure me now, but if my quality of life can be stabilised, I could enjoy some time with my grandson with what’s left.”
Calaneet Balas, President and CEO of the ALS Association, responded to the indecision around the approval of the drug, stating: “We cannot allow perfection to stand in the way of real progress towards turning ALS from a fatal disease into a liveable one. The FDA’s own ALS guidance acknowledges that people with ALS are willing to accept greater risk for the possibility of some benefit.”
Merck under official investigation in France
Merck, a science and technology company based in Germany, is under investigation after “possibly issuing misleading information” regarding its product Levothyrox, used for treating thyroid issues.
Merck changed the formula for Levothyrox in 2017, removing lactose to make it easier to handle. However, this change has been linked to side effects such as memory loss, weight gain and palpitations.
The plaintiffs have accused Merck of not providing sufficient information regarding the change in its product.
Merck has said that the proceedings, taking place in Marseille, France, are not concerned with the product’s new formula, and that it is about the way the company provided information when changing from the old formula to the new.
Mario-Pierre Stasi, the lawyer for Merck, said the company would continue to co-operate with authorities on the matter and that full legal proceedings must be allowed to run their course.
In March 2022, the Cour de Cassation ‒ France’s highest court ‒ confirmed a 2020 Lyon appeal court ruling, which ordered Merck to pay €1,000 in compensation to the 3,300 people suffering with thyroid problems in France, after the 2017 change of formula.