Therapeutic areas in focus
FDA clears GSK’s Boostrix for use in pregnancy
The FDA has approved the use of GlaxoSmithKline’s Boostrix (Tdap) vaccine for use in the third trimester of pregnancy to prevent pertussis (whooping cough) in infants younger than two months old.
According to the Centers for Disease Control and Prevention (CDC), 4.2% of the total cases of pertussis reported in the US in 2021 were in infants younger than six months of age, and approximately 31% required hospitalisation. Boostrix is already used for active booster immunisation in children aged 10 to 18 years against tetanus, diphtheria and pertussis, and has been previously approved for use in pregnancy to protect the mother.
The determination of the effectiveness of Boostrix administered during the third trimester to prevent pertussis among infants younger than two months was based on a re-analysis of Boostrix-relevant data.
108 cases of pertussis in infants younger than two months (including four cases where the mother received Boostrix during the third trimester) and 183 control infants who didn’t have pertussis (including 18 whose mother received Boostrix in the third trimester) resulted in a preliminary estimate of Boostrix being 78% effective.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, stated: “Pertussis disease is a highly contagious respiratory illness affecting all age groups. However, babies are at highest risk for getting pertussis and having serious complications from it. While vaccination is the best method for providing protection, infants younger than two months of age are too young to be protected by the childhood pertussis vaccine series. This is the first vaccine approved specifically for use during pregnancy to prevent a disease in young infants whose mothers are vaccinated during pregnancy.”
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.”