Pharmaceutical Technology Editors

The European Medicines Agency (EMA) has issued its recommendation that Evrysdi be granted marketing authorization in the European Union for the treatment of patients with certain types of spinal muscular atrophy (SMA).

According to a Feb. 26, 2021 press release, the regulatory agency’s decision has been based on two clinical studies, which investigated the effects of Evrysdi on patients with infantile-onset SMA and later-onset SMA. The trial results demonstrated a beneficial effect on the motor development and survival at 12 months in young patients when compared with the natural course of the disease.

Evrysdi is a non-invasive oral treatment that contains the active substance, risdiplam. As a part of the recommendation, EMA’s Committee for Medicinal Products for Human Use (CHMP) has asked Roche to perform a post-authorization efficacy study to further evaluate disease progression in treated patients versus untreated patients.

“Our close partnership with the SMA community has enabled the development of the first ‘at-home’ treatment for SMA in infants, children, and adults with varying levels of disease severity, the majority of whom remain untreated,” said Levi Garraway, Roche’s chief medical officer and head of Global Product Development, in a company press release issued on Feb. 26, 2021. “If approved, Evrysdi would represent a much-needed therapeutic option and we expect it to become the treatment of choice for people living with SMA and their families.”

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EMA has recommended that Evrysdi be granted marketing authorization in the EU for the treatment of patients with certain types of SMA. 

EMA Recommends Oral Treatment for SMA

In a March 1, 2021 press release, the Russian Direct Investment Fund (RDIF) announced that the Sputnik V vaccine for COVID-19 has been approved in Slovakia under the emergency use authorization procedure.

The latest approval marks the second within the European Union for the Russian vaccine and makes Slovakia the 39th country in the world to authorize the use of Sputnik V in coronavirus patients. The Slovakian authorization decision was based on a comprehensive assessment of the vaccine by Slovakian experts and the results of the Russian clinical trials.

“In Europe Sputnik V vaccine has been approved in Slovakia, Hungary, San-Marino, Serbia, Montenegro, Republika Srpska, Belarus, and Russia with other countries of the region also showing strong interest in it,” saidKirill Dmitriev, CEO of RDIF, in the press release. “Vaccine partnership and diversification of national vaccine portfolios are the key for helping people feel safe and protected, restoring economic activity, and ending the pandemic as soon as possible. We have received numerous requests from EU states to provide Sputnik V directly to them based on the reviews of their national agencies. We will continue to do so as well as work with [the European Medicines Agency] based on the rolling review procedure we initiated in January.”

RDIF has announced that the Sputnik V vaccine for COVID-19 has been approved in Slovakia under the emergency use authorization procedure.

Slovakia Approves Sputnik V Vaccine

The European Commission (EC) has launched a structured dialogue aimed at gaining a better understanding of global medicines supply chains and to identify potential causes and drivers of vulnerabilities.

Bringing together actors of the pharmaceuticals manufacturing chain, public authorities, research communities, health professionals, and patient organizations, the dialogue is one of the flagship initiatives of the EC’s Pharmaceutical Strategy, which was adopted in November 2020. A particular focus of the dialogue will be the development of production capacity for critical APIs, raw materials, and medicines in the European Union to ensure better preparedness for future pandemics, and will serve to put forward a set of policy recommendations to address the identified vulnerabilities by the end of 2021.

The launch event was opened by Margaritis Schinas, EC vice-president, with Stella Kyriakides, EC commissioner for Health and Food Safety, and Thierry Breton, EC commissioner, Internal Market, acting as moderators of the roundtable discussion with stakeholders. The Portuguese Presidency was represented by Marta Temido, the country’s minister for Health, and the European Parliament was represented by Dolors Montserrat and Nathalie Colin-Oesterlé—both ministers of the European Parliament.

The EC has launched a structured dialogue aimed at gaining a better understanding of global medicines supply chains and to identify potential causes and drivers of vulnerabilities.

EC Launches Structured Dialogue to Address Medicines Supply Vulnerabilities

API and drug product manufacturers have until March 31, 2021 to assess the risk of nitrosamines in approved or marketed products following FDA’s extension of the recommended timeframe to complete the first of three steps from six to seven months.

On Feb. 24, 2021, FDA announced a revision to its Sept. 3, 2020, guidance for industry, 

Control of Nitrosamine Impurities in Human Drugs

, giving manufacturers more time to complete the first of three steps to detect and prevent unacceptable levels of nitrosamine impurities in pharmaceutical products.

Since 2018, nitrosamines have been found in several commonly prescribed drugs. The guidance recommendations apply to all chemically synthesized APIs, drug products containing chemically synthesized APIs, and drug products at risk due to other factors described in the guidance.

The first step is an assessment of the risk of nitrosamine impurities in APIs, marketed products, and products under approved and pending applications. The risk assessment documents should be retained and be available if requested; the documents do not have to be filed with FDA.

If the manufacturer determines an API or drug product is at risk, they should conduct confirmatory testing for the presence of nitrosamine impurities. Any changes implemented to prevent or reduce nitrosamine impurities in APIs and drug products should be reported to FDA.

FDA moves recommended timeline for API and drug manufacturers to assess impurity risks to March 31, 2021.

Nitrosamine Risk Assessment Date Extended

WuXi AppTec announced on March 2, 2021 that it has acquired OXGENE, a UK-based contract research and development organization focused on the design and development of scalable gene therapy technologies.

Under the terms of the acquisition, OXGENE will become a fully owned subsidiary of WuXi Advanced Therapies (WuXi ATU), WuXi AppTec’s cell and gene therapy contract testing, development, and manufacturing organization (CTDMO) business unit, WuXi Apptec said in a company press release. OXGENE will be WuXi ATU’s first facility in Europe and will work to enhance WuXi ATU’s abilities to transform the development, testing, manufacturing, and commercialization of cell and gene therapy products while accelerating their time to market.

“OXGENE is proud to have invented a range of next-generation platforms that address the complexities of viral vector manufacturing for gene and cell therapies,” said Dr. Ryan Cawood, founder and CEO of OXGENE, in the press release. “We are excited to join the team at WuXi ATU and to work together to scale up our technologies as a combined business, which will strengthen and broaden our service offerings for customers, improving by orders of magnitude the scale and cost of bringing cell and gene therapies to market. The benefits will be significant for the industry and for patients worldwide.”

“We are delighted to welcome OXGENE to WuXi ATU,” added Dr. David Chang, CEO of WuXi ATU, in the press release. “By combining WuXi ATU’s world-class cell and gene therapy CTDMO platform with OXGENE’s innovative capabilities, we will be able to provide transformative solutions for our customers. This business combination represents a significant step in our ongoing effort to enable our customers and partners worldwide to deliver more effective and accessible advanced therapies to patients globally.”

Under the terms of the acquisition, OXGENE will become a fully owned subsidiary of WuXi Advanced Therapies, WuXi AppTec’s cell and gene therapy contract testing, development, and manufacturing organization business unit.

WuXi AppTec Acquires OXGENE to Increase Cell and Gene Therapy Offerings

Takeda announced on Feb. 26, 2021 that it has entered into an agreement to divest four of its diabetes products in Japan to Teijin Pharma, a Japanese pharmaceutical company, for JPY 133 billion (US$1.2 billion).

The portfolio consists of four non-core type 2 diabetes products sold in Japan: Nesina (alogliptin), Liovel (alogliptin/pioglitazone), Inisync, and Zafatek (trelagliptin), Takeda said in a company press release. Takeda is divesting the products because they are outside of Takeda’s current business focus which includes gastroenterology, rare diseases, plasma-derived therapies, oncology, and neuroscience.

“Today’s transaction enables us to sharpen our focus in Japan on developing and delivering highly-innovative products within Takeda’s five key business areas,” said Masato Iwasaki, president of Takeda’s Japan Pharma Business Unit, in the press release. “We look forward to maintaining our seamless collaboration with Teijin Pharma as we continue to manufacture and supply these valued products on their behalf, and to making even greater contributions to our purpose of achieving better health and a brighter future for people in Japan and around the world.”

“This announcement builds on the continued execution of Takeda’s operational and financial commitments of optimizing our portfolio for growth and paying down long-term debt, while delivering life-transforming treatments to patients worldwide,” added Costa Saroukos, chief financial officer at Takeda, in the press release. “With longstanding expertise in type 2 diabetes, we are confident that Teijin Pharma is the right partner to maximize the value of these trusted products and ensure uninterrupted patient access in Japan.”

Takeda is divesting the products because they are outside of Takeda’s current business focus, which includes gastroenterology, rare diseases, plasma-derived therapies, oncology, and neuroscience. 