Health

FDA Clears Drug for Leading Form of Cystic Fibrosis

Published

9 years ago

July 3, 2015

MATTHEW PERRONE, AP Health Writer

WASHINGTON (AP) — Federal health officials have approved a new combination drug for the most common form of cystic fibrosis, the debilitating inherited disease that causes internal mucus buildup, lung infections and early death. But it will come at a steep price — more than $250,000 for a year’s treatment.

The Food and Drug Administration cleared the twice-a-day pill from Vertex Pharmaceuticals Inc. for a variation of cystic fibrosis that affects about 8,500 people in the U.S. who are 12 years and older. The approval notice was posted to the agency’s website Thursday.

The new drug — to be sold as Orkambi — is Vertex’s follow-up to its breakthrough pill Kalydeco, which became the first drug to treat the underlying cause of cystic fibrosis in 2012. Orkambi combines Kalydeco with a new drug ingredient, lumacaftor.

Kalydeco is only approved for a cluster of rare cystic fibrosis forms that affect about 2,000 patients who are 2 years old and up.

About 30,000 Americans live with cystic fibrosis, which is caused by variety of genetic mutations passed from parents to their children. The disease causes sticky mucus to buildup in the lungs and other organs, which leads to infections, digestive problems and eventually death.

Vertex said Orkambi will cost $259,000 per year. That’s less than the $311,000 annual price tag for Kalydeco.

Dr. Robert Giusti of New York University’s Langone Medical Center noted that half of all U.S. cystic fibrosis patients have the form targeted by Orkambi, which occurs when a child inherits two copies of a certain genetic mutation — one from each parent. He expects the FDA will eventually expand the drug’s approval to patients as young as 6, increasing the number of people who could benefit.

“This is really exciting because this is a disease that causes a 1 to 2 percent deterioration each year in lung function of patients,” said Giusti, who directs the center’s cystic fibrosis program. “Now they have a therapy available to potentially reverse that effect.”

In the 1950s, children with cystic fibrosis seldom survived long enough to complete elementary school. Due to improvements in care, the typical cystic fibrosis patient today can expect to survive into their early 40s, according to the Cystic Fibrosis Foundation.

Prior to the approval of Kalydeco, drugs used to treat cystic fibrosis focused on controlling symptoms — such as opening up lung airways and breaking up mucus. Kalydeco was the first drug to target the underlying genetic defect that causes the disease.

Expectations for Vertex’s new drug have been tempered by study results that, while statistically significant, were not as dramatic as those first reported with Kalydeco. In company trials, patients treated with Orkambi for six months reported a 2.5 to 3 percent improvement in lung function, a key measure for cystic fibrosis patients. That improvement met the FDA’s standards for effectiveness, but did not equal results seen with Kalydeco, which improved lung function by about 10 percent.

Company officials have pointed out the differences in the forms of the disease targeted by the two drugs. Kalydeco was developed for patients who have a problem with a protein on their cell walls, which doesn’t properly balance the flow of water and salt from the cell. Orkambi targets patients with two problems — the protein does not reach the cell wall and, once there, does not work properly.

Shares of Vertex rose $5.07, or 4 percent, to close at $131.26 in trading Thursday. Its shares are up almost 34 percent over the past year.

Vertex’s cystic fibrosis drugs grew out of a long-term partnership with the Cystic Fibrosis Foundation. Vertex Pharmaceuticals Incorporated received roughly $120 million in research and funding from the foundation, culminating in the 2012 approval of Kalydeco. The twice-a-day pill had sales of $464 million last year, according to the Cambridge, Massachusetts company.

Last November the Cystic Fibrosis Foundation sold its royalty rights to Kalydeco and Orkambi for $3.3 billion.

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Mind, Body, and Spiritual Well-Being for Women Addressed in NAACP Forum in Oakland

The Women In The NAACP Oakland Branch is proud to announce the upcoming “Total You – Mind, Body, and Spirit Women’s Health Forum” scheduled for April 27 at Acts Full Gospel Church. Running from 9 a.m.-2 p.m. at 1034 66th Ave., this forum aims to provide an empowering platform for women to engage in discussions, gain knowledge, and access resources pertaining to their health and well-being.

Published

5 days ago

April 20, 2024

Oakland Post

The panel will address various aspects of women’s health, including physical, mental and emotional well-being and relationships. iStock photo by Jorge Elizaquibel.

Special to The Post

The Women In The NAACP Oakland Branch is proud to announce the upcoming “Total You – Mind, Body, and Spirit Women’s Health Forum” scheduled for April 27 at Acts Full Gospel Church.

Running from 9 a.m.-2 p.m. at 1034 66^th Ave., this forum aims to provide an empowering platform for women to engage in discussions, gain knowledge, and access resources pertaining to their health and well-being.

The forum will feature renowned experts, healthcare professionals, and advocates from Genentech, John Muir Health, Sutter Health of The East Bay, Kaiser Permanente, and the Alameda County Public Health Department.

Our expert panel will address various aspects of women’s health, including physical, mental, and emotional well-being, and healthy relationships. The forum will encompass a wide range of topics such as breast cancer, menopause, reproductive health, nutrition, mental health awareness, preventive care, and much more.

Participants will have the opportunity to attend informative sessions, interactive workshops, and panel discussions led by experts in their respective fields. Additionally, there will be wellness activities, screenings, and informational booths offering valuable resources and support.

This forum is open to women of all ages and backgrounds, encouraging inclusivity and diversity in the conversation surrounding women’s health. Whether you’re seeking information for yourself, a loved one, or simply looking to connect with other women, this event promises to be enlightening and empowering.

For more information and to register for the Total You Women’s Health Forum, please visit https://www.naacpoakland.org/ or contact Dr. Delores Thompson. WIN chairwoman at (510) 328-3638.

The Women In The NAACP Oakland Branch is dedicated to empowering women, and young teen girls. We look forward to your participation in this important event.

To register, go to https://www.naacpoakland.org/events/the-total-you-womens-health-forum

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Alameda County

Oakland Conducts Its Biennial ‘Point in Time’ Homelessness Count

Oakland, along with other cities in Alameda County, conducted their biennial ‘Point In Time’ census count on Feb. 1 to gain a thorough understanding of the size and dispersion of the homeless population in the region. The Point In Time (PIT) count is federally required by the Housing and Urban Development Department as a requirement to receive funding and resources to tackle homelessness in the area.

Published

6 days ago

April 20, 2024

Oakland Post

Abandoned vehicles on 24th and Wood St where volunteers conducted the count and surveys. Photo by Magaly Muñoz.

By Magaly Muñoz

The Point In Time (PIT) count is federally required by the Housing and Urban Development Department as a requirement to receive funding and resources to tackle homelessness in the area.

David Modersbach, Grants Manager of Alameda County Health Care for the Homeless program, said that the methodology this time around was different, as this count had a much more personal “lived experience” aspect that previous counts did not have.

In 2022, the county relied more on statistical extrapolation and assumptions, but this year’s survey questionnaires allowed for details on substance abuse issues, how long someone has been living without proper housing, what resources people are in need of and much more.

“[The PIT count is] a critical opportunity for the county, Continuum of Care, and cities to understand the magnitude of homelessness in Alameda County. [The count] enables us to better allocate resources and implement effective programs to tackle this issue head-on in a compassionate and inclusive way,” Modersbach said.

St. Mary’s Center was one of the many meeting hubs across the county that hosted volunteers and community officials the morning of the count. The organization has been deeply involved in the effort to provide resources for unhoused people and others in need.

St. Mary’s is a nonprofit in West Oakland that helps seniors and preschool families with food and housing. Last year, the organization helped about 50 seniors find housing after they had fallen on hard times.

Sharon Cornu, executive director of St. Mary’s, said a lot of the older couples and individuals that come into the center have borne the brunt of the skyrocketing cost of living in the Bay Area. The most recent influx of seniors St. Mary’s has seen coming in for help has been made up of people who were evicted when the COVID-19-related moratorium on rent payment ended.

“Seniors are the fastest growing segment of the unhoused and the incredibly high cost of housing is driving them to the streets,” Cornu said.

Among the volunteers were workers with Operation Dignity, a nonprofit organization that helps veterans and those living on the street find shelter, transitional housing and supportive services.

“These are our stomping grounds,” Ivan Magana, program manager for Operation Dignity said.

Magana stated that his team was extremely familiar with the people residing in the encampments they were conducting the count in since Operation Dignity made many visits to these areas while doing community outreach. He said they had even informed some of the unhoused people they knew about the count a few days prior so they would not be alarmed when the enumerators showed up early in the morning to conduct the count.

Not everyone got the memo though, as the volunteers encountered an almost violent situation around the 6 a.m. when a young woman living in a bus yelled at the Operation Dignity workers to leave her alone.

Luckily, the three-year experience Mangana has working with Operation Dignity and his knowledge of therapeutic health services, equipped him with the techniques needed to deescalate the tension. The woman soon realized who the volunteers were and apologized, he said.

Another volunteer and Operation Dignity worker, Yolanda Kirkpatrick, noted that she was initially hesitant because of the early schedule. She felt the time deterred others from participating, too.

Her prediction would come true as the hours went on and they continued to walk along 24th St in downtown Oakland and there was very little activity on the streets.

The volunteers shared similar sentiments. Although the community the people they were engaging for the count and surveys encounter tend to distrust outsiders, the PIT count was necessary for the city to receive the appropriate level of federal funds to address a crisis that is spiraling out of control in California.

A full analysis and report of the count will be made available in the summer.

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Community

For Cervical Cancer Month, Medical Community Focused on Education

January was Cervical Cancer Awareness Month. Physicians, advocates and others in the medical community commemorated the month by raising awareness about a form of cancer they say is highly preventable and treatable. Cervical cancer is caused by a virus called the human papillomavirus (HPV) and it develops slowly over time but can be prevented with proper care in girls as young as 13 years old.

Published

1 week ago

April 19, 2024

Oakland Post

A Mayo Clinic article published last month stated that Black women are more likely to be diagnosed and die of cervical cancer, compared to White women in the U.S. 2,000 Black women are diagnosed every year with cervical cancer and 40% die as a result.

By Magaly Muñoz

January was Cervical Cancer Awareness Month.

Physicians, advocates and others in the medical community commemorated the month by raising awareness about a form of cancer they say is highly preventable and treatable.

Cervical cancer is caused by a virus called the human papillomavirus (HPV) and it develops slowly over time but can be prevented with proper care in girls as young as 13 years old.

Sonia Ordonez, an OBGYN and gynecology surgeon at Kaiser Permanente, stated that as soon as people with cervixes reach the maturity reproductive age, they should start taking preventative measures like getting the HPV vaccine. The vaccine involves a series of two-doses for people aged 9 through 14 or three-doses for people 15 through 45 years old.

“I see a lot of young women who can’t remember or may not have gotten [the vaccine] when they were younger, or maybe got one, but we can give them the series of vaccines and restart at any point in time,” Ordonez said.

She said that cervical cancer is not the only cancer caused by HPV. Strains of the virus can also lead to throat, anal and penile cancers.

Screening is also an effective way to check for cervical cancer and should be done every three years after someone turns 21, doctors recommend. It is best to start as early as possible to catch occurrences early.

Ordonez said that this cancer is also more likely found in people of color and has led to more deaths overall.

A Mayo Clinic article published last month stated that Black women are more likely to be diagnosed and die of cervical cancer, compared to White women in the U.S.

2,000 Black women are diagnosed every year with cervical cancer and 40% die as a result.

“This disparity is not due to genetic differences among White, Black or Hispanic women, but rather related to systemic racism, access to healthcare and socioeconomic factors,” Dr. Olivia Cardenas-Trowers, a Mayo Clinic urogynecologist, said in the article.

Ordonez stated that immigrant women are also highly susceptible to the cancer, as many Latin American countries may not have accessibility to screenings or lack of insurance makes it harder for them to get tested.

Hispanic women are 40% more likely to be diagnosed with cervical cancer, and 30% more likely to die from it, as compared to non-Hispanic White women, according to the Office of Minority Health.

Family medicine physician, Joy Anyanwu, stated that the pandemic contributed to hesitancy about getting cervical cancer screenings among some women. Other factors are people’s aversion to vaccines, parents not wanting to believe that their children are or will become sexually active, and doubt about the overall effectiveness of the vaccine.

“The vaccine is very safe — over 97% effective in preventing cervical cancer,” Anyanwu said. “Even if you aren’t having sex, the earlier you start would actually help.”

Anyanwu said she understands that parents might not want to ask questions about their children’s reproductive health, but it’s a mindset that can be a barrier to having important conversation about prevention or care.

To keep families their families and communties healthy, the doctor emphasized that people should prioritize keeping up with their vaccine series and going to screenings every year.