This Week at FDA: Update on ORA revamp, new drug approvals, and more

Welcome to another installment of This Week at FDA, your weekly source for updates – big and small – on FDA, drug and medical device regulation, and what we’re reading from around the web. This Week FDA updated its proposal to create a new food program and revamp its Office of Regulatory Affairs (ORA) in the process, approved a slew of new products, and issued several guidances.

Possibly the biggest regulatory news of the week is the US Food and Drug Administration's (FDA) gave an update on how it proposes to create a unified Human Foods Program (HFP), which includes a new model for ORA. The agency is proposing that the new Deputy Commissioner for Human Foods have oversight of all budget and resource allocations for the entire HFP, including ORA resources; compliance functions currently managed within ORA would be merged into the HFP and the product centers' existing compliance functions; and certain functions under the Office of Security and Emergency Management, currently in the Office of Operations, would be transitioned to ORA.

STAT News that a year after Gilead and Johnson & Johnson sued drug wholesaler Safe Chain Solutions for its alleged role in distributing counterfeit HIV pills, FDA warned the same company for failing to ensure that “suspect” medicines did not enter the marketplace.

Earlier this month, FDA sent the wholesaler a letter rebuking it for violating the Drug Supply Chain Act. The violations include doing business with unauthorized trading partners, failing to maintain proper recording-keeping and failing to respond to a notification of an illegitimate product.

After Merck, Bristol Myers Squibb and the US Chamber of Commerce filed a lawsuit against the US government arguing a CMS guidance on negotiating Medicare prices that included a confidentiality provision violated their constitutional rights, the US government has revised the guidance to allow drug companies to publicly discussion pricing negotiations at their discretion.

A closely-watched abortion lawsuit to ban the abortion pill mifepristone may hinge on whether the Texas-based anti-abortion doctor who brought the suit has legal standing. While the case has focused on FDA's safety and approval process of the drug, the person bringing the case has to show they have suffered harm or face an imminent injury traceable to the defendant, which in this case is FDA.

Drugs & Biologics

This week FDA officials gave an update on the agency's Oncology Quality, Characterization, and Assessment of Real-world Data (QCARD) Initiative meant to improve the quality of initial cancer study proposals. The agency says insights from the initiative have helped inform information requests sent by oncology review teams when they have received real-world data (RWD) study proposals that lack sufficient information.

The Center for Biologics Evaluation and Research (CBER) published their list of guidances that they plan to publish this calendar year. The agency notes its list includes topics that currently have no guidance associated with them, topics where updated guidance may be helpful, and topics for which CBER has already issued Level 1 draft guidances that may be finalized following review of public comments.

FDA has published a final guidance entitled “Chronic Rhinosinusitis With Nasal Polyps: Developing Drugs for Treatment” to help sponsors developing drugs or biological to treat chronic rhinosinusitis with nasal polyps (CRSwNP). It outlines the agency's thinking on trial population and design, effectiveness, statistical analysis, and safety of drugs for the treatment of CRSwNP.

FDA has also published a draft guidance on the prohibition on wholesaling under Section 503B of the Federal Food, Drug, and Cosmetic (FD&C) Act. The guidance outlines the agency's interpretation of, and policies concerning, the prohibition on wholesaling under the law and describes examples of how it intends to apply the provision.

FDA plans to hold a meeting of its Cardiovascular and Renal Drugs Advisory Committee on 13 September to discuss Alnylam Pharmaceuticals' drug Onpattro (patisiran) to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults.

A majority of FDA’s Endocrinologic and Metabolic Drugs Advisory Committee this week voted to that Ipsen Biopharmaceuticals’ drug palovarotene to treat the ultra-rare disease fibrodysplasia ossificans progressiva has proven to be effective and its benefits outweigh the risks.

FDA sent a Form 483 to Indian drugmaker Ipca Laboratories citing it for eight violations. Agency inspectors found issues with the machinery and manufacturing process, and said the company failed to respond to the issues adequately.

Roche subsidiary Genentech is pulling the thyroid cancer drug Gavreto (pralsetnib) from the US market because it's not feasible to get full approval of the drug according to its partner Blueprint Medicines. While Gavreto has FDA accelerated approval to be on the market for patients over the age of 12 with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, Genentech would have had to prove the drug is clinically beneficial in confirmatory trials to get full approval.

FDA has approved Pfizer's NGENLA (somatrogon-ghla), a once-weekly human growth hormone to treat patients three years and older who have growth failure due to inadequate secretion of endogenous growth hormone according to the company. The decision was based on results from a non-inferiority Phase 3 trial that compared the NGENLA against to the human growth hormone somatropin. Pfizer says the drug will be available in the US this August.

BioMarin's adeno-associated virus vector-based gene therapy drug Roctavian (valoctocogene roxaparvovec-rvox) was approved by FDA to treat adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test. The drug is administered as a single dose by intravenous infusion and consists of a viral vector carrying a gene for clotting Factor VIII.

FDA has also CellTrans' Lantidra, the first allogeneic pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes. It is approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin (average blood glucose levels) because of current repeated episodes of severe hypoglycemia (low blood sugar) despite intensive diabetes management and education.

UCB's Rystiggo (rozanolixizumab) to treat adults with generalized myasthenia gravis (gMG) who have antibodies against the acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK) proteins also received FDA approval. The agency also approved Pfizer's Litfulo (ritlecitinib) as the first drug to treat severe alopecia areata (AA) for patients as young as 12 years old where their hair loss is caused by an autoimmune disease.

Medtech

FDA made a formal announcement this week of its intention to use the rule-making process to regulate laboratory-developed tests (LDT) via the Unified Agenda. While the agency has used regulatory discretion to refrain from regulating the tests, it has said it will develop a rule to regulate them unless Congress passes a new law to create a new LDT regulatory framework.

AgencyIQ also noticed FDA has held at least one meeting this month with members of the American Clinical Laboratory Association (ACLA) which has opposed LDT regulation in the past to discuss diagnostics regulatory reforms.

The Center for Devices and Radiological Health (CDRH) has listed several enhancements to its Electronic Medical Device Reporting (eMDR) system on its website that is used by manufacturers to report medical device adverse events. FDA says its eSubmitter client is updated concurrently with the eMDR system, but industry with system-to-system, or AS2, accounts with the FDA Electronic Submissions Gateway (ESG), should use the information on its website to plan system updates to align with the eMDR system enhancements as soon as possible.

FDA announced the recall of the ARROW Endurance Extended Dwell Peripheral Catheter System which is intended to allow health care providers access to a patient’s peripheral vascular system, for short term use, to sample blood, monitor blood pressure, or administer fluids, blood, and blood products. The agency said Teleflex, and their subsidiary Arrow International, are recalling the devices after reports of catheter separation or leakage which may cause serious injury, heart attack, or death.

Roche has gotten FDA clearance for another pair of assays that can help diagnose Alzheimer’s. The Elecsys Beta-Amyloid (1-42) CSF II, or Abeta42, and Elecsys Total-Tau CSF, or tTau, assays can run on any of Roche’s Cobas immunoassay analyzers, and can help measure the concentration of beta-amyloid and tau proteins, that are linked to the development of Alzheimer’s.

This Week at FDA: Update on ORA revamp, new drug approvals, and more

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