Vertex Pharmaceuticals announced a plan late Tuesday to begin trials before the end of the year of the third in its so-called “triple combo” of pills designed to treat as much as 90 percent of the 75,000 patients worldwide who suffer from cystic fibrosis. That news, announced in conjunction with the Boston-based drugmaker’s third-quarter financial results last night, spurred a 6 percent stock increase after hours, implying the company’s market cap could increase by about half a billion dollars…

Wednesday’s initial public offering for Cambridge-based Ra Pharmaceuticals marked the ninth biotech startup to go public this year, tying the number in 2013 but still less than either of the two years since. Ra (Nasdaq: RARX), which has 40 employees in one of the former Pfizer buildings in Alewife, ended up with the third-largest IPO size for any Massachusetts-based biotech in 2016, with a total of $92 million raised from the sale of 7 million shares for $13 each. That’s more than the $86 million…

On Friday, 21 emerging medical device companies will present their technologies and business plans to a group of local investors at the annual MedTech Showcase, hosted by the Massachusetts Medical Device Industry Council. More than 300 venture leaders and business leaders are expected to attend the event tomorrow, Oct. 28 from 8 a.m. to 2 p.m. at the Westin Waltham, 70 Third Ave. As a main event, John McDonough, president and CEO of Lexington-based T2 Biosystems (Nasdaq: TTOO), will be interviewed…

Editor's note: This story was originally published Friday morning, and has been updated to reflect the FDA's decision regarding the drug later that day. A U.S. approval decision for a major drug planned to be marketed by Cambridge-based Sanofi Genzyme that had been expected last Friday has been delayed due to “deficiencies” found during a manufacturing site inspection in France. In its third quarter report, released Friday morning, French drugmaker Sanofi (NYSE: SNY) disclosed that “manufacturing…

Third Rock Ventures, the Boston-based venture capital firm behind some of the Bay State’s most prominent biotechs, has reclaimed its title as the biggest life science-focused VC firm in the state with a new $616 million round, and has also named its first female partner in eight years. With the announcement of its Fund IV today — its largest ever — the firm now has raised $1.9 billion in the nine years since it was formed. That eclipses its rival across the Charles River, Flagship Ventures,…

Today, Shire plc confirmed it won’t make any new investments from Baxalta Ventures, the short-lived venture capital arm of the drug company Shire acquired in June.

The results of a 33-patient study conducted by a Burlington biotech suggest its long-acting steroid injection for osteoarthritis of the knee may be safer for the large percentage of those patients who also have type 2 diabetes. Flexion Therapeutics (Nasdaq: FLXN) has for years been developing its lead drug candidate, Zilretta (formerly called FX006), a reformulation of a common corticosteroid that’s used with osteoarthritis patients. Flexion’s version combines the drug with a employs proprietary…

On Thursday, Harvard University will open a 15,000-square-foot life science lab in Allston named after Steve Pagliuca, and executive at Bain Capital and co-owner of the Boston Celtics. The Pagliuca Harvard Life Lab will be the home to 20 startup ventures founded and run by Harvard faculty, alumni, students, and postdocs. The first 17 of those were revealed by the university a couple weeks ago, and they include drug and vaccine developers as well as DNA sequencing companies. Mayor Marty Walsh will…

Latest innovation in quadrivalent meningococcal vaccination designed for use in persons 2 years of age and older in the U.S.

• Independent Data Monitoring Committee recommended continuing ongoing Phase 3 trial only in the more advanced “critical” group with Kevzara higher-dose versus placebo and discontinuing less advanced “severe” group

- Libtayo decreased the risk of death by 32.4% compared to chemotherapy

Objective responses seen in 29% of patients with locally advanced basal cell carcinoma (BCC)

Do we ever learn from our past mistakes? For many years we believed that technology was an inevitable force for good. It would give us instant access to a near infinite amount of information and allow us to easily and instantly connect with nearly anyone on earth. What could go wrong? The answer is that...

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US-based drugmaker Mylan and partner India-based biologicals specialist Biocon have announced the launch of their pegfilgrastim biosimilar, Fulphila, in Australia. The drug can be used to treat neutropenia (a lack of white blood cells) in cancer patients.

There are major regulatory lapses in the manufacturing of similar biologics in India. The use of scientific audits could strengthen the regulatory system and improve the provision of high quality biosimilars in the country, according to a recent opinion piece [1] by Dr GR Soni, which was published in GaBI Journal.

The US Food and Drug Administration (FDA) has approved the first generic version of a commonly used inhaler, marketed as Proventil, and the anti-parasitic Daraprim, which has previously been the subject of a price gouging scandal.

Celltrion has submitted an application to the European Medicines Agency (EMA) for its adalimumab biosimilar, currently known as CT-P17.

An online educational course has been published by Medscape in collaboration with the Association of Diabetes Care & Education Specialist.

In response to the COVID-19 pandemic, authorities in Australia and the European Union have allowed drug producers to collaborate to ensure medicine production and supply.

Taiwanese biosimilars developer Mycenax announced on 28 April 2020 that it had made a deal with Hungary-based Gedeon Richter (Richter) regarding its tocilizumab biosimilar.

Biological drugs remain unaffordable for many in the US due to strategies used by pharmaceutical companies and negative messaging about biosimilars, explains a recent commentary by Dr Joel Lexchin, School of Health Policy and Management at York University, Canada [1].

On 7 April 2020, China’s Center for Drug Evaluation (CDE) published draft guidance on clinical trials for the approval of bevacizumab copy biologicals. This guidance is the second specific guideline released by the CDE in April. The agency also released guidance on adalimumab on 1 April 2020 [1].

US-based drugmaker Mylan and partner, India-based biologicals specialist Biocon, announced on 28 April 2020 the launch of their pegfilgrastim biosimilar, Fulphila, in Canada. This is the second biosimilar from the pair to be launched in the country.

Top-line: The Congressional schedule and work practices are uncertain over the next few months, leaving many questions about whether and how appropriations bills will move forward. There is a possibility that increased funding at public health agencies, including FDA, may be exempt from FY 21 budget caps. The Alliance’s meeting with Dr. Boon, FDA Associate Commissioner […]

The Alliance met (virtually) on April 15 with Dr. Caitlin Boon, FDA Associate Commissioner for Food Policy and Response and several of her colleagues. She described CFSAN’s and CVM’s role in responding to the current pandemic, as well as how the agency is assuring a continued safe food supply. About 50% of food spending in the […]

Top-Line: There is no new information about the appropriations schedule for Congress. The House will require a rules change for appropriators to work remotely. The death of former FDA Commissioner Donald Kennedy is noted. More on FDA’s extraordinary efforts to address food safety and supply issues. Eventually, Congress will need to address FY 21 funding and […]

Q: Congress must act on FY 21 appropriations. What are the possible ways for Congress to address this? A: Congress must decide if they will do substantive work on the 12 appropriations bills with the goal of passing full-year funding bills before October 1. One alternative would be to fund the beginning of the fiscal year […]

Top-Line: This week we look at House and Senate schedules for returning to DC and starting on the FY 21 appropriations process. This week’s Analysis and Commentary explores the reasons why advances in science and technology foretell that FDA will need significant additional funding in FY 21. This column focuses on vaccine development. Senate Returns […]

FDA — along with NIH, CDC, and other front-line public health agencies — is caught up in the urgent COVID-19 efforts. Appropriately, enormous resources are being devoted to fighting the pandemic and more funding will come, if needed. At the same time, we are getting positive reports on the FDA’s efforts to carry out the […]

Top-line: Congress is inching forward on the FY 21 appropriations process. However, neither the House nor the Senate has adopted rules permitting virtual committee mark-ups. Also hanging over the process is the tight budget ceiling for non-defense discretionary (NDD) programs. Various ways it might be solved and implications for FDA are discussed in this week’s Analysis […]

Congress is starting to consider ways to address the budget cap problem that hangs over the entire FY 21 appropriations process for non-defense discretionary (NDD) programs. Last year, Congress broke a long-running stalemate by agreeing to budget caps for FY 20 and FY 21. They decided to front-load the increases, making spending decisions (relatively) easier […]

WASHINGTON, D.C. – Co-Chairs of the Childhood Cancer Caucus, Reps. Michael McCaul (R-TX), Jackie Speier (D-CA), Mike Kelly (R-PA), and G. K. Butterfield (D-NC), applaud the passage of the Senate companion to their bill – S. 292, the Childhood Cancer STAR Act.

The STAR Act passed the House today by a unanimous vote.  It addresses the four major concerns facing the pediatric cancer community: Survivorship, Treatment, Access, and Research, and will elevate and prioritize the fight against childhood cancer at the National Institute of Health (NIH).

The members released the following joint statement:

"Today was a long anticipated day for the pediatric cancer community, and one to be celebrated.  This bill is the most comprehensive childhood cancer bill to ever pass Congress and will finally head to the president’s desk to be signed into law. Childhood cancer remains one of the deadliest killers of our kids and we as a Congress, and a nation, must say, ‘Enough is enough.’  As co-chairs of the Childhood Cancer Caucus, we would like to thank all those who made this possible, including the Alliance for Childhood Cancer and the entire childhood cancer advocacy community.”

Click here to watch McCaul’s floor remarks ahead of the House passage of the STAR Act.

WASHINGTON, D.C. – Five lawmakers introduced a bipartisan bill giving a full range of medical services to families with children who have life-limiting illnesses and who qualify for Medicaid, which currently has gaps in such coverage.

The Children’s Program of All-Inclusive Coordinated Care (ChiPACC) Act (H.R. 6560) would let states create comprehensive care programs for these children. Its authors are the Co-Chairs of the Congressional Childhood Cancer Caucus: Representatives Michael McCaul (R-TX), Jackie Speier (D-CA), G.K. Butterfield (D-NC), and Mike Kelly (R-PA), together with Representative Diana DeGette (D-CO), a senior member of the House Energy and Commerce Committee.

“Families with children facing life-limiting illnesses need all the support they can get, and they should be empowered to seek out that support,” the bill’s sponsors said in a joint statement. “We owe it to these kids and their loved ones to help ensure more compassionate care in their most trying times.”

Gaps in Medicaid coverage of hospice and palliative services have deprived many beneficiaries of the care they need because the program does not cover some of children’s unique medical needs.

Under this bill, the family of every child who qualifies for Medicaid will receive a specialized care plan covering a range of services – palliative, counseling, respite, expressive therapy and bereavement – providing them and their families greater comfort and peace of mind.

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Rick Bright confirmed as having left his position as director of BARDA as the agency provides aid to develop solutions for COVID-19.

The facility will be the companyâs second facility in Carlsbad specifically for its BioReliance viral and gene therapy service.

Sangamo plans to utilize Mogrifyâs cell conversion technology to develop CAR-Treg cell therapies.

J&J agrees a manufacturing partnership with Emergent, as it looks to hit its target of one billion doses.

Takeda in global licensing agreement with ProThera to develop plasma-based therapies for inflammatory conditions.

AskBio acquires gene therapy biotech working on treatments for neurodegenerative disorders.

The company receives approval for MenQuadfi to prevent meningococcal disease.

The WHO and world leaders commit to a fund to accelerate development of vaccines, tests and treatments for COVID-19.