Pfizer's VELSIPITY Granted FDA Approval for Moderate to Severe Active Ulcerative Colitis in Adults

Pfizer has received the green light from the U.S. Food and Drug Administration (FDA) for VELSIPITY, also known as etrasimod, an oral medication that is taken once daily and selectively modulates the sphingosine-1-phosphate (S1P) receptor.

 This approval is specifically for adults who are dealing with moderately to severely active ulcerative colitis (UC), a chronic and often disabling condition affecting an estimated 1.25 million individuals in the United States. 

The recommended dose for 2 mg Symptoms of UC can be distressing and encompass chronic diarrhea with blood and mucus, abdominal pain, and a compelling sense of urgency, and the impact goes beyond the physical aspect due to the chronic and unpredictable nature of the symptoms. 

 The FDA approval was grounded on the promising outcomes of the ELEVATE UC Phase 3 registrational program, specifically the ELEVATE UC 52 and ELEVATE UC 12 trials. These trials evaluated the safety and efficacy of VELSIPITY 2 mg once daily in inducing clinical remission among UC patients who had previously failed or were intolerant to at least one conventional treatment, biologic, or Janus kinase (JAK) inhibitor therapy. Notably, a significant proportion of patients in these trials were treatment-naïve to biologic or JAK inhibitor therapy. 

The studies achieved all primary and key secondary efficacy endpoints, while maintaining a safety profile consistent with previous VELSIPITY studies. 

 In the ELEVATE UC 52 trial, patients treated with VELSIPITY demonstrated a substantial increase in clinical remission rates compared to those on placebo at both week 12 and week 52. at both week 12 and week 52 Similarly, in the ELEVATE UC 12 trial, a noteworthy proportion of patients on VELSIPITY achieved clinical remission compared to the placebo group at week 12. These findings, along with positive outcomes in key secondary endpoints like endoscopic improvement and mucosal healing, substantiated the efficacy of VELSIPITY in treating UC. VELSIPITY represents a significant advancement as a once-daily, oral medication that selectively binds with specific S1P receptor subtypes. 

Regulatory applications for VELSIPITY's approval in treating ulcerative colitis have been submitted in various countries worldwide, including Canada, Australia, Mexico, Russia, Switzerland, and Singapore. The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for VELSIPITY, and a decision from EMA is expected in the early months of 2024. 

The pivotal ELEVATE UC 52 trial was designed with a 12-week induction period followed by a 40-week maintenance period, employing a randomized, double-blind, placebo-controlled approach. The primary goal was to assess the safety and efficacy of etrasimod 2 mg once daily on clinical remission after both 12 and 52 weeks. The trial demonstrated significant improvements in primary and key secondary endpoints, including endoscopic improvement and mucosal healing. 

 Similarly, the ELEVATE UC 12 trial, which was also randomized and placebo-controlled, focused on assessing the efficacy and safety of etrasimod 2 mg once daily in subjects with moderately to severely active UC. The trial achieved its primary objective, showcasing a noteworthy proportion of patients achieving clinical remission with etrasimod compared to the placebo group at week 12 at week 12 

Importantly, the safety profile of VELSIPITY was consistent across both trials, with common adverse reactions being manageable and not unexpected. These trials affirm that initiating VELSIPITY treatment does not necessitate a complex up-titration regimen, enhancing its practicality and ease of use for patients

FDA Ad Comm Votes Unanimously in Favor Of COMIRNATY Booster for Emergency Use in People 65+ and Certain High-risk Populations

 Pfizer (NYSE: PFE) and BioNTech SE (Nasdaq: BNTX) have announced that the U.S. Food and Drug Administration’s (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously to recommend the FDA grant Emergency Use Authorization (EUA) for a booster dose of COMIRNATY (COVID-19 Vaccine, mRNA) in individuals 65 years of age and older and individuals at high risk of severe COVID-19. 

The committee recommended that the additional dose be administered at least six months after the two-dose series. The panel also agreed that healthcare workers and others at high risk for occupational exposure should be included in this EUA.

VRBPAC based its recommendation on the totality of scientific evidence shared by the companies, including data from their clinical program evaluating the safety, tolerability and immunogenicity of a booster dose of COMIRNATY. A booster dose of the vaccine elicited significantly higher neutralizing antibody titers against the initial SARS-CoV-2 virus (wild type), as well as the Beta and Delta variants, when compared with the levels observed after the two-dose primary series. The reactogenicity profile within seven days after the booster dose was typically mild to moderate, and the frequency of reactions was similar to or lower than after dose two. The adverse event profile was generally consistent with other clinical safety data for COMIRNATY.

Real-world surveillance data also were presented to the VRBPAC by the Israel Ministry of Health, providing further support for the public health impact of boosters. The data presented from Israel included an analysis published this week in The New England Journal of Medicine. The analysis comprised approximately 1.1 million individuals ages 60 years and older who were eligible for a booster dose of the vaccine between July 30 through August 31, 2021. No new safety signals were observed, and reported adverse events were lower than those observed after dose two. The analysis showed that a booster dose restored very high levels of protection against COVID-19 infections and severe disease in this period when Delta was the dominant strain. Individuals who received the booster dose were less likely by a factor of 11.3 (95% CI: 10.4, 12.3) to develop a confirmed infection and less likely by a factor of 19.5 (95% CI: 12.9, 29.5) to develop severe illness compared to those who were previously fully vaccinated but did not receive a booster dose.

China halts imports of Pfizer drug on paperwork glitch

China suspended imports of U.S. drugmaker Pfizer Inc's AIDS-related drug Diflucan on Tuesday, citing a problem with late paperwork, the country's food and drug watchdog said in a statement on its website.

Pfizer, the largest drugmaker in the United States, contravened Chinese law when one of its France-based factories failed to submit a supplementary application on time, the China Food and Drug Administration (FDA) said in the statement.

With the country's healthcare spending forecast to nearly triple to $1 trillion by 2020 from $357 billion in 2011, according to consulting firm McKinsey, China is a magnet for makers of medicines and medical equipment.

Pfizer has taken steps to resolve the issue and is working with China's FDA to ensure its products comply with Chinese law, it said in a statement on its Chinese-language website. The issue is not linked to quality or safety, it added.

Some analysts said the incident looked like a paperwork "glitch" and should be short-lived.

"It should not impact too much Pfizer's business in China and I am sure the imports will be resumed once the procedure is complete," said Simon Li, Shanghai-based general manager at Kantar Health China.

China has been cracking down this year on the healthcare sector, with investigations ranging from allegations of corporate bribery to how drugs are priced, as well as drives to increase quality and safety levels across the sector.

Diflucan, which treats fungal infections linked to AIDS, had worldwide sales of $259 million in 2012, according to Pfizer's latest annual financial statement, a small fraction of the firm's $59 billion revenue that year. Pfizer has more than 9,000 employees in China and operates in more than 250 Chinese cities

~Reuters

Pfizer to stop using Blackberry phones

AllThingsD reports that pharmaceutical company Pfizer has advised all of its BlackBerry-using employees to move on to iOS or Android smartphones for work due to BlackBerry’s current “volatile state.” A Pfizer spokesperson explained to AllThingsD that it was recommending that employees continue to ”use their BlackBerry devices and… migrate to a new device at normal contract expiration.” Pfizer employs around 90,000 people and AllThingsD’s sources say the company’s BlackBerry deployment is “huge.” There is a silver lining for Pfizer workers who don’t want to give up their BlackBerrys, however — they’ll at least be able to get BBM on their new iPhones and Android phones

Source: BGR

Virtual Trials - Still REMOTE ?

Just as I wrote about virtual trials (interestingly came to know about it around the "Partnership in Clinical Trials" conference where there was a session on this topic), there was an update on this topic supporting my initial apprehensions of the practicality of this fascinating approach. 

Miguel Orri, Senior Director of clinical sciences at Pfizer, at the ‘Partnerships in Clinical Trials’ conference, have indicated that the virtual trial "REMOTE" did not recruit.

Reasons:

• It did not ‘appreciate what patients need’ - Patients don’t want to divulge their medical information online, or to a pharma company.
• Another problem is that many patients affected by overactive bladder disorder are elderly, and do not use the internet as regularly as younger patients. (I've seen many granny's are on FaceBook these days.. shouldn't be a problem in the near future)
• Importantly, the aspects of the process were ‘quite complicated and tedious

Plan of Action from Pfizer:

Pfizer is collecting feedback from participants in the US trial to improvise the model. They have also now taken steps to revamp the model by creating a new call centre to helps patients through the initial steps. 

This bitter start in US should help them with their European virtual trial, dubbed REMOTE 2.0. Pfizer said they would contract a recruitment vendor to help patients through the initial setup process, making it easier for patients. All participants in the European trial will also receive a communication device to help with updating their e-diaries.

source: InPharm

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Virtual Clinical Trials - Pfizer leads the race

Virtual Clinical Trials - Pfizer leads the race

I was very much interested to know what this is all about when I saw the Program of "Partnerships in clinical trials" conference that started yesterday at The Marriot World Cener, Orlando, FL, USA. Really interesting idea.

How about running clinical trials at a subject's home ?! Yes, that is all about Virtual Clinical Trials.

Pfizer have become the forerunner of this new approach in clinical trials. They have decided to leverage the advantages that the digital world provides to overcome the difficulties faced while conducting a usual clinical trial. Difficulties range from finance, logistics, partnering, enrolling patients, labs, ethical and legal issues, storage, dispensing of IP, etc. in a clinical trial. If not all the difficulties, many of those seems to be overcome in virtualtrials (which has limitations of its own!)

Pfizer began the first ever virtual clinical trial, REMOTE (Research on Electronic Monitoring of OAB Treatment Experience) to determine whether the results of the pilot ‘virtual trial’ can replicate those of a previously completed Phase IV study with tolterodine tartrate (Detrol LA)

• 600 patients from around 10 states across the US
• Screening is online
• Consent online using video/multimedia
• Subjects will manage their own trial activity and report results directly to a trial investigator,
• Study drugs will be shipped to the subjects' homes rather than to study sites
• Study participants will get $25 for each online assessment and/or laboratory visit completed, up to a total of US$175.
• No study site visits

Potential advantages would be real time and reliable data collection, a lot of time, money and effort saved. A lot of paperwork is eliminated and of course audit findings will be clear.

“Studies like REMOTE could make biomedical science much more accessible to people who have long been excluded from or under-represented in clinical trials,” said Dr Freda Lewis-Hall, executive vice president and Chief Medical Officer of Pfizer.

But to train almost 600 people on the systems to use, procedures to follow will be tough job for Pfizer and the advent of technology is itself a limitation of its use. And the main disadvantage of this would be that this system is more applicable to conditions where there are a lot of patient reported outcomes and those that does not involve any complex diagnostic or treatment procedures. That again raises a concern of the scalability of this system. Moreover, I cannot imagine something like this to be a reality (at least in half a decade) in an African country or a developing nation where the cost of healthcare is still a economical burden on the patients.

Least, this does spice up the clinical trial experience for trial personnel and subjects who are tech savvy!

Here is the program at "Partnerships in Clinical Trials" confernence. No wonder Pfizer dominates this  session. They lead the race with no visible competitors.

Monday, March 5, 2012—Executive Summit Day and General Session Kick-Off 9:30 Chairperson's Opening Remarks,PFIZER INC. 9:45 Virtual Trials— what are They? - PFIZER INC. 1030 Making a Study Different: What is Truly Innovative About the Virtual Trial Approach? - GENENTECH (ROCHE) 11:00 Break 11:30 Case Study: New Results From Pfizer's Mobile- Powered Virtual Clinical Trial Outsourcing Pilot Program, PFIZER INC. 12:30 Lunch 1:30 Assess the Legal Risks and Considerations of Virtual Trials, GORDON & REES LLP. 2:15 Examine Collaboration of Technology Companies Needed to Support the Virtual Trial Model 2:45 Industry Collaboration: How Should We Approach and Work With the FDA to Create a Regulatory Process for Virtual Trials? - PFIZER INC. AND JANSSEN 3:30 Summits End