FDA withdraws approval of preterm birth drug Makena
The US Food and Drug Administration (FDA) has rescinded its approval for Swiss drug maker Covis Pharma's (Covis) Makena drug and its generic versions, the only approved treatments aimed at reducing the risk of premature birth.
The decision was made after studies did not prove the effectiveness of the drug in preventing preterm birth. Makena was approved by the FDA under the accelerated approval pathway in 2011 to reduce the risk of preterm birth in women with a singleton pregnancy who have a history of spontaneous preterm birth.
The approval came with a condition that a confirmatory clinical trial should be undertaken to evaluate the predicted clinical benefit to newborns. The results of the trial neither reduced preterm births nor demonstrated any improvement in the health of newborn babies. Last month, the company announced that it was voluntarily pulling the drug off the market in response to the Presiding Officer’s report recommending the withdrawal of the approval for the drug.
Vertex and CRISPR Therapeutics submit BLAs to FDA for exa-cel
Vertex Pharmaceuticals and CRISPR Therapeutics have completed the rolling submission of biologics licence applications (BLAs) to the US Food and Drug Administration (FDA) for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
The applications include requests for priority review. If granted, these would shorten the time taken for application review to eight months. Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. It is being assessed for the treatment of SCD or TDT patients.
The BLAs are supported by data obtained from the ongoing Phase III CLIMB-111 and CLIMB-121 trials, along with an ongoing long-term follow-up CLIMB-131 trial. The CLIMB-111 and CLIMB-121 trials are designed to evaluate the efficacy and safety of a single exa-cel dose in TDT or SCD patients aged 12 to 35 years.
The US FDA granted regenerative medicine advanced therapy (RMAT), orphan drug, fast track, and rare paediatric disease designations to exa-cel for the treatment of SCD and TDT.
Gennova applies for Covid-19 booster emergency use authorisation in India
Gennova Biopharmaceuticals has applied for an emergency use authorisation (EUA) from the Drug Controller General of India (DCGI) for its mRNA-based Omicron-specific Covid-19 booster vaccine.
The firm, which is a partner of PharmaJet, has submitted interim safety and immunogenicity endpoints data from the Phase II/III trial to the Indian regulator to secure the EUA.
Pharmajet stated that Gennova’s booster is the first in India for treating the Omicron variant. Dubbed GEMCOVAC-OM, the needle-free vaccine will be delivered intradermally with the PharmaJet Tropis Precision Delivery System (PDS).
The lyophilised vaccine remains stable at 2°C-8°C and can be distributed using the current refrigeration supply chain across India, along with other low and middle-income countries (LMICs).
Unlike other approved mRNA vaccines, GEMCOVAC-OM need not be stored in ultra-low temperature environments.
FDA tackles opioid crisis with OTC Narcan approval
On March 29, the US Food and Drug Administration (FDA) granted approval for the first nonprescription, “over-the-counter” (OTC) naloxone nasal spray, Narcan. Emergent BioSolutions’ Narcan (naloxone hydrochloride) is a medicine used to reverse opioid-related overdoses.
This approval comes at a time when the US is facing a growing opioid crisis. The overdose death rate in the US increased more than 250% from 1999 to 2019, as per a Canada-US joint white paper on substance use and harms. Between 1999 and 2021, overdose deaths from synthetic opioids excluding methadone increased 97-fold, and prescription opioid overdose deaths increased 4.9-fold, with fentanyl overdoses being a major problem.
The FDA first approved Narcan 4mg nasal spray as a prescription drug in 2015. More recently, in November 2022, the FDA announced that its preliminary assessment of a selection of naloxone products, such as Narcan, showed certain prescription naloxones had the potential to be safe and effective for OTC use. OTC availability of the drug is expected by late summer.
Moderna to set up new mRNA manufacturing facility in Kenya
Moderna has finalised an agreement with the government of the Republic of Kenya to establish an mRNA manufacturing facility in the country. The company will construct the new advanced mRNA facility, which is claimed to be Moderna’s first mRNA manufacturing facility in Africa and is expected to have the capacity to produce up to 500 million vaccine doses annually.
The new facility is expected to enable the manufacturing of drug products for Kenya and Africa, and will be able to quickly scale up its production and respond to public health emergencies on the continent and worldwide. The company is also committed to establishing mRNA manufacturing facilities in Australia, Canada, the US and the UK.
Kenya's investments, trade, and industry cabinet secretary Moses Kuria said: “This investment creates the momentum to meet the $10bn annual target under the government's manufacturing '20 by 30' vision, where we plan to grow the contribution of manufacturing to GDP to 20% by the year 2030 from the current 7%.”