The National Consumers League (NCL) is urging the FDA to commit to a transparent, patient-centered process before it makes a final decision about withdrawing Amag Pharmaceuticals Inc.’s Makena (hydroxyprogesterone caproate injection) and its generics from the U.S. market.

Removal of the drug, commonly known as 17P, would leave at-risk pregnant women and their providers without an approved standard of care to reduce the risk of preterm birth. The group noted that COVID-19 may increase the risk of preterm birth. “Especially during this pandemic, pregnant women and their unborn babies are under extreme stress, yet providers have few therapeutic options to help at-risk mothers,” the group said in an Oct. 7 statement.

The statement came two days after the FDA proposed withdrawing Makena from the market because the required postmarketing study didn’t show clinical benefit. The agency gave Amag and the sponsors of the generics 15 days to request a hearing on the proposal, but that doesn’t mean it has to grant one.

Amag, of Waltham, Mass., said it’s considering its options. If a hearing is granted, the process could take months, during which time Makena and the approved generics would remain available to patients.

In June, 14 health care providers and consumer, women’s health and maternal health organizations joined with the NCL in sending a letter to the FDA to express “serious concerns that a regulatory decision could be based on a single study that was largely conducted outside of the U.S., in a predominantly white population,” the NCL said.

“In our outreach to the FDA, we urged the agency to consider alternative ways to further evaluate and define the patient populations that most benefit from 17P, without depriving women of access. We regret to state that the agency did not respond to our letter, nor [to] two separate requests to meet with stakeholders,” the NCL said.

According to the NCL statement this week, Amag submitted a proposal to the FDA earlier this year with a plan for a retrospective study to identify predictors of the drug’s benefit in women with a history of recurrent preterm birth, but the company was not given an opportunity to discuss the plan with the agency.

The NCL noted that 59% of the participants in the single phase III study used to win accelerated approval in 2011 were African American women. However, African Americans represented 7% of a predominantly international patient population used in the PROLONG confirmatory trial, which showed no statistically significant difference between the Makena and placebo arms in improving the gestational age of delivery or reducing adverse neonatal outcomes from preterm birth.

Health Canada explains COVID-19 disclosures

Health Canada released guidance Oct. 7 explaining its public release of clinical information for COVID-19 drugs and devices imported and sold in Canada under interim orders.

While personal and confidential business information will be protected, safety and efficacy information will be made publicly available after Health Canada completes its regulatory review process, according to the guidance.

The safety and efficacy information will be automatically disclosed in applications submitted under the interim order for importing, selling and advertising drugs and disclosed upon request in applications submitted under the order for importing and selling medical devices.

In addition to keeping Health Canada’s decision-making transparent, public access could provide “valuable information that may help with the use or development of COVID-19 drugs and medical devices,” the agency said.

TGA on changing sponsor details

Australia’s Therapeutic Goods Administration (TGA) issued guidance Oct. 8 on changing sponsor details in the product information (PI) and labels for prescription drugs.

The guidance applies to situations in which there is a change in the sponsor or the existing sponsor changes its name or address. Changes in sponsor details must be made in the PI immediately after the change occurs. They must be reflected in the label within 12 months, according to the guidance.

A new sponsor of a registered prescription medicine must notify the TGA of the change in sponsorship and apply for a variation to the Australian Registry of Therapeutic Goods (ARTG) entry. The ARTG must reflect the new sponsor’s details before an application can be made to change the drug’s PI or labels.

FDA awards research grants for rare diseases

The FDA said Oct. 8 that it has awarded a total of more than $16 million in six new clinical trial research grants through its Orphan Products Grants Program.

“These important clinical trials are at a critical time where additional resources are needed to support rare disease research during the COVID-19 pandemic," FDA Commissioner Stephen Hahn said.

The six were chosen from 47 applications by more than 90 rare disease and clinical trial experts from industry and academia. The grant program is intended to support clinical studies of products that address unmet needs in rare diseases or conditions or that provide highly significant improvements in treatment or diagnosis.

The awards went to researchers at:

  • Acucela Inc., of Seattle – $1.6 million over three years for a phase III study of emixustat hydrochloride for the treatment of Stargardt disease;
  • The Fred Hutchinson Cancer Research Center, of Seattle – $3.5 million over four years for a phase II study of ustekinumab (Stelara, Johnson & Johnson) for the prevention of graft-vs.-host disease;
  • Seattle Children's Hospital – $3 million over four years for a phase Ib study of I.V. gallium nitrate for the treatment of cystic fibrosis patients colonized with nontuberculosis mycobacterium;
  • State University of New York Stony Brook – $3.1 million over four years for a phase I study of CD4 redirected CAR T therapy for the treatment of CD4-positive T-cell neoplasms;
  • University of Cincinnati – $1.9 million over four years for a phase I/II study of ABTL-0812, Barcelona-based Abiltypharma SL’s Akt/mTOR inhibitor, for the treatment of pancreatic cancer;
  • University of Virginia, of Charlottesville, Va. – $3.2 million over four years for a phase II study of oral azacytidine plus romidepsin for the treatment of peripheral T-cell lymphoma.

Se habla español

As part of its ongoing effort to educate patients and health care providers about biosimilars, the FDA posted a Spanish version of its biosimilars patient webpage.

It also published Spanish versions of infographics explaining the basics of biosimilars, as well as a biosimilars consumer update to better inform patients and providers about biologics and their relationship to biosimilars, including interchangeables. The materials discuss the concepts of safety and effectiveness, as well as the benefits of using biosimilars.

The FDA’s effort to educate patients and providers has helped address the skepticism many people in the U.S. have about biosimilars, Sheila Frame, vice president of marketing, market access & patient services at Sandoz Inc., told BioWorld. That effort has made a big difference in the market uptake of biosimilars, she said, adding that “education is really, really important.”

No Comments